[House Hearing, 108 Congress]
[From the U.S. Government Printing Office]
ASSESSING DIGESTIVE DISEASES RESEARCH AND TREATMENT OPPORTUNITIES
=======================================================================
HEARING
before the
SUBCOMMITTEE ON HEALTH
of the
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED EIGHTH CONGRESS
SECOND SESSION
__________
JULY 8, 2004
__________
Serial No. 108-94
__________
Printed for the use of the Committee on Energy and Commerce
Available via the World Wide Web: http://www.access.gpo.gov/congress/
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------------------------------
COMMITTEE ON ENERGY AND COMMERCE
JOE BARTON, Texas, Chairman
W.J. ``BILLY'' TAUZIN, Louisiana JOHN D. DINGELL, Michigan
RALPH M. HALL, Texas Ranking Member
MICHAEL BILIRAKIS, Florida HENRY A. WAXMAN, California
FRED UPTON, Michigan EDWARD J. MARKEY, Massachusetts
CLIFF STEARNS, Florida RICK BOUCHER, Virginia
PAUL E. GILLMOR, Ohio EDOLPHUS TOWNS, New York
JAMES C. GREENWOOD, Pennsylvania FRANK PALLONE, Jr., New Jersey
CHRISTOPHER COX, California SHERROD BROWN, Ohio
NATHAN DEAL, Georgia BART GORDON, Tennessee
RICHARD BURR, North Carolina PETER DEUTSCH, Florida
ED WHITFIELD, Kentucky BOBBY L. RUSH, Illinois
CHARLIE NORWOOD, Georgia ANNA G. ESHOO, California
BARBARA CUBIN, Wyoming BART STUPAK, Michigan
JOHN SHIMKUS, Illinois ELIOT L. ENGEL, New York
HEATHER WILSON, New Mexico ALBERT R. WYNN, Maryland
JOHN B. SHADEGG, Arizona GENE GREEN, Texas
CHARLES W. ``CHIP'' PICKERING, KAREN McCARTHY, Missouri
Mississippi, Vice Chairman TED STRICKLAND, Ohio
VITO FOSSELLA, New York DIANA DeGETTE, Colorado
STEVE BUYER, Indiana LOIS CAPPS, California
GEORGE RADANOVICH, California MICHAEL F. DOYLE, Pennsylvania
CHARLES F. BASS, New Hampshire CHRISTOPHER JOHN, Louisiana
JOSEPH R. PITTS, Pennsylvania TOM ALLEN, Maine
MARY BONO, California JIM DAVIS, Florida
GREG WALDEN, Oregon JANICE D. SCHAKOWSKY, Illinois
LEE TERRY, Nebraska HILDA L. SOLIS, California
MIKE FERGUSON, New Jersey CHARLES A. GONZALEZ, Texas
MIKE ROGERS, Michigan
DARRELL E. ISSA, California
C.L. ``BUTCH'' OTTER, Idaho
JOHN SULLIVAN, Oklahoma
Bud Albright, Staff Director
James D. Barnette, General Counsel
Reid P.F. Stuntz, Minority Staff Director and Chief Counsel
______
Subcommittee on Health
MICHAEL BILIRAKIS, Florida, Chairman
RALPH M. HALL, Texas SHERROD BROWN, Ohio
FRED UPTON, Michigan Ranking Member
JAMES C. GREENWOOD, Pennsylvania HENRY A. WAXMAN, California
NATHAN DEAL, Georgia EDOLPHUS TOWNS, New York
RICHARD BURR, North Carolina FRANK PALLONE, Jr., New Jersey
ED WHITFIELD, Kentucky BART GORDON, Tennessee
CHARLIE NORWOOD, Georgia ANNA G. ESHOO, California
Vice Chairman BART STUPAK, Michigan
BARBARA CUBIN, Wyoming ELIOT L. ENGEL, New York
JOHN SHIMKUS, Illinois GENE GREEN, Texas
HEATHER WILSON, New Mexico TED STRICKLAND, Ohio
JOHN B. SHADEGG, Arizona DIANA DeGETTE, Colorado
CHARLES W. ``CHIP'' PICKERING, LOIS CAPPS, California
Mississippi CHRIS JOHN, Louisiana
STEVE BUYER, Indiana BOBBY L. RUSH, Illinois
JOSEPH R. PITTS, Pennsylvania JOHN D. DINGELL, Michigan,
MIKE FERGUSON, New Jersey (Ex Officio)
MIKE ROGERS, Michigan
JOE BARTON, Texas,
(Ex Officio)
(ii)
C O N T E N T S
__________
Page
Testimony of:
Carron, Adam................................................. 21
DeRose, Rodger, President and CEO, Crohn's and Colitis
Foundation of America...................................... 17
Peura, David, Digestive Disease National Coalition........... 24
Spiegel, Allen M., Director, National Institute of Diabetes
and Digestive and Kidney Diseases.......................... 11
Additional material submitted for the record:
Peura, David, Digestive Disease National Coalition, response
for the record............................................. 38
(iii)
ASSESSING DIGESTIVE DISEASES RESEARCH AND TREATMENT OPPORTUNITIES
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THURSDAY, JULY 8, 2004
House of Representatives,
Committee on Energy and Commerce,
Subcommittee on Health,
Washington, DC.
The subcommittee met, pursuant to notice, at 11:05 a.m., in
room 2322, Rayburn House Office Building, Hon. Michael
Bilirakis (chairman) presiding.
Members present: Representatives Bilirakis, Upton, Buyer,
Barton (ex officio), Brown, Pallone, Capps, and Rush.
Also present: Representative Kelly.
Staff present: Cheryl Jaeger, majority professional staff;
Eugenia Edwards, legislative clerk; and John Ford, minority
counsel.
Mr. Bilirakis. Good morning.
Today's hearing assessing digestive disease research and
treatment opportunities addresses an important issue that is
often, unfortunately, overlooked.
Digestive diseases such as ulcers, heartburn, celiac
disease and inflammatory bowel disease, IBD, effect
approximately 70 million Americans. Some of these diseases are
minor and easily manageable while others are debilitating and
extremely painful. All are increasing, unfortunately, in their
frequency.
One example of IBD, Crohn's disease, was once a very rare
disorder but today there are twice as many cases as there were
30 years ago, and we are not sure why.
Now, it is common to hear the personal stories about how
IBD's affect an individual's family or a close friend. Members
of my own family, for example, suffer from IBD.
What also troubles me greatly is that these conditions are
increasingly afflicting children and teenagers. The health of
our children is already in crises, especially with the
skyrocketing obesity rates. Children with digestive diseases
often have to live with symptoms such as pain, intestinal
bleeding and weight loss. And that is why I am so honored to
welcome on of our witnesses here today, Adam Carron who will
speak about his personal experience with Crohn's disease. Adam,
really no one should have to endure what you have experienced
over the years with this terrible illness. You have my
gratitude and my admiration and that of my colleagues for
speaking out and educating others on Crohn's disease. It is not
easy to discuss the terrible ordeals you have gone through, and
we certainly thank you so very much for being here today.
We have an excellent panel of witnesses here this morning.
In addition to Adam, I would like to welcome Dr. William
Spiegel, Director of the National Institute of Diabetes and
Digestive and Kidney Disease, NIDDK, within the National
Institutes of Health.
NIDDK is the lead institute responsible for the strategic
planning and priority setting of federally funded research in
digestive diseases. NIDDK already has highlighted digestive
diseases and specifically IBD as a priority. And we are, of
course, very happy to know that. In fact, NIDDK has a center
for the study of inflammatory bowel disease and a center for
gastrointestinal biology and diseases which focus on IBD
research.
In the 108th Congress, this subcommittee has held 5
hearings to highlight research activities and budget and
priority setting at the NIH. Both Chairman Barton and I have
expressed our commitment to reauthorize the NIH and assisted
Director of NIH, Dr. Elias Zerhouni to implement his NIH
Roadmap for Medical Research. During this process I am working
to ensure that NIDDK has the resources it needs to maintain its
reputation of excellence in the digestive diseases field, but
also increases the transparency in the way that research is
disseminated to the public. After all, what good is research if
it is not in some good way disseminated to the public.
I am also excited to have representatives from two
digestive disease patient advocacy groups. Rodger DeRose is the
President and CEO of the Crohn's and Colitis Foundation of
America, CCF. And Dr. David Peura is the Associate Chief in the
Division of Gastroenterology and Hepatology and Professor of
internal medicine at the University of Virginia Health Science
Center. Mr. Peura is testifying on behalf of the Digestive
Disease National Coalition, DDNC.
I would like to commend both of your organizations,
gentlemen, on your work to improve education, access to and
quality of digestive disease health care for current and future
patients.
I cannot stress enough how dedicated I am to increasing
research and education for digestive diseases. I was fortunate
to able to speak to CCF last month and I was touched by the
level of commitment there that there is in the effort to
fighting these debilitating diseases. Many Members of Congress
are concerned about digestive diseases, and leading that group
is our colleague and friend, Congresswoman Sue Kelly and then
additionally Congressman Roy Blunt and Bobby Rush have
introduced bills to increase the focus on digestive diseases.
Ms. Kelly is not a member of this committee, but at my
invitation and I know without any objection from Mr. Brown or
the other members, she is here today and is sitting up here as
a member of the panel, and we certainly welcome her.
I believe it is important that we work together to find a
cure and ways to prevent these very serious diseases. At the
end of the day I know most of our concern is that if we do not
find a solution, the prevalence of digestive diseases will
continue to grow.
Again, thank you for being here today.
And I gladly yield to the ranking member of the
subcommittee, my good friend from Ohio Mr. Brown for an opening
statement.
Mr. Brown. Thank you, Mr. Chairman. Thank you all the
witnesses for joining us today.
The term digestive disorder covers a wide range of
illnesses, as the chairman said, several of them deadly, all of
them significant from a public health perspective. Liver
disease and inflammatory bowel disease or IBD, acid reflux,
cancer of the stomach and pancreas, of these illnesses and many
more are considered digestive disorders. More than 62 million
Americans are diagnosed with one of these illnesses every year.
I was contacted by Paul Levin, who is the father of Sarah,
a young woman with Crohn's disease. Crohn's is a form of IBD,
it is incurable and it is insidious. It causes inflammation of
the lining of the intestine. When inflamed, the lining becomes
ulcerated and bleeds. It can cause severe diarrhea, abdominal
pain, cramping, fever and rectal bleeding.
Sarah Levin takes 11 medications everyday. She has major
surgery. She has taken steroids that stunted her growth and
compromised her physical health. She has been forced to miss
work again and again because Crohn's can flare up at anytime.
Regrettably there is no cure at this time. Medical
treatment can only try to ease the pain and control the
inflammation.
Like my digestive disorders, IBD does not get the attention
it deserves given its prevalence and its disabling effects.
Approximately 1 million Americans suffer from inflammatory
bowel disease, and numbers keep increasing as there are about
twice as many cases today of IBD as there were 30 years ago.
Sarah and I hope all of us are determined to do about this.
Along with her father and other advocates she has been lobbying
very successfully for legislation focusing on IBD. H.R. 290
sponsored by Sue Kelly and be Jessie Jackson, Jr. would raise
public awareness about IBD, jump start IBD related research and
help IBD patients get the treatment that they need. I am
pleased to support this bill.
I am interested in learning more about H.R. 3756 which
would establish a national commission on digestive disorders. A
commission was formed many years ago in 1976 to get a better
handle on the prevalence of these diseases and the nature and
score of available treatments. Obviously medicine has evolved
significantly over these past three decades. The demographics
of our nation and relevant lifestyle factors such as diet and
exercise have also changed over time. If congressional action
is needed to update the original commission's findings in
regard to digestive disorders, reinstating the commission is a
step Congress should consider.
I would add, Mr. Chairman, as you know the Bush
Administration budget memo recently leaked to the Washington
Post revealed the Administration's intent to cut overall
funding for NIH after the election, I might add, by roughly
$600 million for fiscal year 2006. If anything, this hearing
has demonstrated that our national investment in medical
research is more important than ever. We have no business
cutting NIH. In fact, we frankly have no business in slowing
down its growth and its increased spending, which this Congress
has already done. And next year if President Bush gets his way,
it will be much more.
We have, as I said, witnessed a slowdown in funding
increase and this proposed cut that the President has suggested
is absolutely going in the wrong direction. I hope, Mr.
Chairman, we can learn more about that during this hearing.
Thank you.
Mr. Bilirakis. The Chair yields to the gentleman from
Indiana, Mr. Buyer.
Mr. Buyer. I just want to note for the record, sometimes I
find it breathless. The record is very clear that when
Republicans took over Congress we set out as a goal, and it was
to double the funding of NIH.
So I almost find it breathless whenever I hear someone ``My
God, Republicans may be cutting NIH.''
Mr. Brown. Would the gentleman yield?
Mr. Buyer. Mr. Brown, I just want to make clear, I have not
seen this document but it makes it even better when you would
testify to--you know, and I thank and appreciate the leadership
the Republicans have done to double NIH funding as we push the
bounds of science that improve the quality of life of people in
our country. That is a good thing.
Mr. Brown, I yield to you.
Mr. Brown. Yes. Thank you, Mr. Buyer.
I only mention that that initiative was originally
President Clinton's, that the Congress then worked with in
partnership bipartisanally, and now that President Bush has
decided to make room----
Mr. Buyer. What initiative was Bill Clinton's?
Mr. Brown. Well, the initiative of doubling the NIH budget,
which began in----
Mr. Buyer. Boy, that is----
Mr. Bilirakis. Regular order. These are opening statements.
We are not debating.
Mr. Buyer. I will just reclaim my time, because I am not--I
do not revitalize history. I do not rewrite history.
Mr. Bilirakis. Let us keep our eye on the ball.
Mr. Buyer. No, no. I----
Mr. Bilirakis. Now all these good people have come here
because they feel that we can work together for a good purpose.
And then we get into partisanship----
Mr. Buyer. Mr. Chairman, yes, we can work together for good
purpose.
Mr. Bilirakis. Well, I would repeat that I was a part of
that promise that was made that we would double NIH funding----
Mr. Buyer. That is right.
Mr. Bilirakis. [continuing] over the next 5 years.
Mr. Buyer. That is right.
Mr. Bilirakis. So I do not know whether President Clinton
had it in his mind to do so, whatever the case may be. I know
it was the Congress that made that particular version, and we
fulfilled it. We do not fulfill too many of the promises we
make up here, unfortunately, but that is certainly what we
will--we did do and we should be proud of it.
But I do not think we should be going into partisanship or
President this or President that. Let us stick to what we are
supposed to be doing here.
Mr. Buyer, please continue, sir.
Mr. Buyer. Well, part of what we are doing here is trying
to improve society and take care of our people. And when
private industry do not make certain investment, and if we can
partner with government, NIH to do that, we all benefit. And
that is what all that is about.
I just want you to know that I just feel uncomfortable when
we have got a very good hearing and people take shots; and that
is what we just had here. So I just felt the need to correct
the record.
I appreciate the witnesses being here today. And this is a
very important issue. And Chairman is right, this is an issue
that does not seem to get a lot of attention; you do not read
on the headlines but it affects a lot of people in our society.
And we appreciate you being here today.
Mr. Bilirakis. The Chair recognizes Mr. Pallone for an
opening statement.
Mr. Pallone. Thank you, Mr. Chairman, for holding this
hearing. And as a member of the House Digestive Disease Caucus,
I understand that these types of disorders are often
overlooked. And my colleague, Representative Sue Kelly and all
the members of the Caucus, have been working hard to raise
awareness of these digestive diseases in Congress. And I
appreciate that as members of the Health Subcommittee we are
today affirming our commitment to recognize digestive diseases
and confront their challenges.
I wanted to take a moment to specifically mention Crohn's
disease. Mr. Brown has discussed the disabilities related to
Crohn's disease, and so I will not repeat them. And I do not
necessarily want to get into this argument about what President
Bush is going to do, but I do mention that there is in fact a
memo that we have expressing the fact that if he is reelected,
that we will see cuts in NIH. And I hope that my Republican
colleagues with us in opposing those. But I am not surprised if
some members on the other side have not seen the document
because I am sure that the Administration does not want anybody
to know about it.
Crohn's disease affects an estimated 1 million Americans.
Many victims of Crohn's are children and, unfortunately, for
these children afflicted with the disease Crohn's becomes a
constant feature of their lives. And I trust we will be
learning much more about this and other disorders from our
expert witnesses. But my point today is that Crohn's disease is
about more than just numbers and biological dysfunction, as I
learned a number of years ago when I met with a remarkable
young man named Gideon Sofer. Gideon is a constituent of mine
from Highland Park, New Jersey and a victim of Crohn's disease.
When I met him he was 16 years old, and I listened to his story
about the years it took him to get a proper diagnoses and how
little is still known about treating Crohn's disease. Despite
all of the medical and emotional obstacles Crohn's has thrown
his way, Gideon has refused to sit ideally by. He founded the
IBD Cure Foundation which works to raise awareness about
digestive disorders.
In addition, he was the leading inspiration behind HCON
resolution 455, a resolution I recently introduced that
advocates from a commemorative stamp to increase awareness of
and research for Crohn's. And Gideon has created an online
petition for the stamp, which has garnered over 4,000
signatures in just a short time.
Since I have been privileged to know Gideon Sofer, this
battle for Crohn's awareness has struck a personal cord with
me. But the reality is that this disorder and other diseases
have always been personal because health care is about real
people and real families who have been dealing with these
diseases with no understanding of the cause or cure. We must
work together to spread awareness, increase research and
improve care. And it is for this reason that I have proposed
HCON res. 455, and I hope that members of this subcommittee
will also lend their support to legislation such as H.R. 290
introduced by Sue Kelly which expands research for IBD. I
believe these types of legislation represent a step in the
right direction.
And, again, I just want to thank the chairman and our
ranking member for supporting this issue. I think this
subcommittee and the Digestive Disease Caucus to work together
in removing some of the harm and enormous burden placed on
millions of Americans suffering from digestive diseases.
Thank you. Mr. Chairman.
Mr. Bilirakis. I thank the gentleman.
Mr. Upton for an opening statement?
Mr. Upton. Thank you, Mr. Chairman. I am going to put my
full statement in for the record. I just want to say a couple
of things.
I helped spearhead the bipartisan effort to double the
funds for the NIH over a 5 year span, a successful effort. And
I can assure anyone listening out there that there is going to
be no cut in the NIH. President Bush is absolutely committed to
increased funding for the NIH, and we have to thank folks like
Chairman Young on Appropriations and Chairman Bilirakis and
Chairman Tauzin, and now Chairman Barton for their support for
the NIH as well. And let me just dispel any myth out there that
the NIH is going to get cut. It ain't going to happen. My mom
always told me it ain't right to say ain't, but it ain't going
to happen.
I also want to welcome Sue Kelly. I am a co-sponsor of H.R.
290. It is something that needs to happen.
I have a dear friend that suffers with Crohn's disease. I
am anxious to see us get a cure and to extend her lifespan and
make sure that she and so many others that suffer from that
disease have a great future ahead of them instead of one that
is a dark shadow.
And I appreciate the efforts of Chairman Bilirakis to have
this hearing. I look forward to being engaged and see
legislation perhaps come out of it. And, obviously, increase
the incentives and the encourage the NIH in their great work.
And I yield back my time.
Mr. Bilirakis. I thank the gentleman.
Ms. Capps for an opening statement?
Ms. Capps. Thank you, Mr. Chairman, for holding this
hearing, a very important hearing. And I want to salute our
colleague and welcome Sue Kelly, and thank you for your
leadership in the legislation.
A particular thanks to our witnesses for appearing today,
especially Mr. Carron. Yours is a very important testimony that
is significant for us to have on the record here in Congress.
So thank you for being here.
I did not prepare an opening statement. I look forward to
the testimony.
Thank you.
Mr. Bilirakis. I thank the gentlelady.
By the way, the opening statement of all members of this
committee have been a part of the record, obviously.
Let us see, Mr. Rush. Mr. Rush, who is co-author of one of
the pieces of legislation regarding these diseases. Bobby, you
are recognized.
Mr. Rush. Thank you, Mr. Chairman.
And to members of the subcommittee, let me begin by
commending the chairman and my good friend Mr. Bilirakis and
the ranking member for their participation in this hearing and
for the outstanding work. And I want to thank you for convening
this hearing.
I have been long concerned about the devastating toll that
digestive diseases and disorders have taken on our country.
Each year more than 62 million of our citizens are diagnosed
with a wide range of gastrointestinal disorders ranging from
the inflammatory bowel disease to acid reflux disease, to GI
cancers; just to name a few of them.
A study conducted by the Lewin Group in 2000 concluded that
the direct and indirect costs of just 17 of these diseases
exceeds $41 billion, however the human pain and suffering
associated with these conditions are enormous and defy
quantifying.
In the State of Illinois the screening rates for the most
serious forms of GI diseases is low, and as a result the
mortality and morbidity rates from these diseases are much too
high.
I am encouraged by the progress that have been made in some
areas of GI research and treatment. Yet, on the other hand, I
find it disheartening that with respect to many of these
afflictions we still lack even a basic understanding of the
causes and the treatments of these dreaded diseases.
I am convinced that what we need is an energized and
coordinated focus to digestive diseases research. And to this
end, Mr. Blunt and I have introduced H.R. 3756, the National
Commission on Digestive Diseases Act of 2004, which we urge the
committee to favorably consider. This bill will direct the
Secretary of HHS to convene a national commission on digestive
diseases to be composed of the leading scientists in the field,
physician specialists who treat patients in hospitals, clinics
and offices everyday, patients who suffer from these
afflictions and the National Institute of Health officials who
manage the digestive disease research portfolio. This
commission will survey the state of GI research and develop a
long range plan with detailed recommendations by areas of
research policy and programmatic development. The commission
would issue its report within 18 months of it enactment.
There is precedent for what we are proposing. A little more
than 25 years ago this committee approved the bill which
created the original National Digestive Diseases Commission.
The Commission's report issued in 1979 laid the foundation for
countless important research developments and breakthroughs in
the areas of digestive diseases.
Mr. Bilirakis. Please summarize, Mr. Rush.
Mr. Rush. I sure will, Mr. Chairman.
But the work of this first Commission serves as a
precedent, the successor national commission on digestive
diseases will galvanize the government, the research community
and the public to undertake a comprehensive and cost effective
campaign to end the scourge of digestive diseases.
Mr. Chairman, I look forward to the hearing today. I
welcome the witnesses. And I look forward to affirmative action
by this committee on the bill that Mr. Blunt and I introduced
in the near future.
Thank you, and I yield back.
Mr. Bilirakis. Well, I assure you we will have affirmative
action regarding this issue and trying to do something about
it. I cannot assure that we will have affirmative action
strictly on your piece of legislation.
Mr. Rush. I am really disappointed----
Mr. Bilirakis. But as you and I discussed yesterday, I
would like to include you in all the discussions that we have.
In any case, without objection I would yield 3 minutes to
Ms. Sue Kelly who has the principal piece of legislation that
has been introduced for some time for an opening statement.
Ms. Kelly. Thank you so much, Mr. Chairman. I thank you for
the opportunity to be with you today.
As the sponsor of H.R. 290, the Inflammatory Bowel Disease
Act, I greatly appreciate your leadership in convening today's
hearing which will bring much needed attention and awareness to
a disease that has long been in the shadows of society.
Crohn's disease, an ulcerative colitis collectively known
as inflammatory bowel disease are chronic disorders of the
gastrointestinal track that cause severe pain and suffering in
the more than 1 million Americans who are affected. As you
pointed out, Mr. Chairman, the IBD can cause severe abdominal
pain, fever, bleeding, ulcerations and chronic diarrhea. But
there are complications as well that are related to this
disease; arthritis, osteoporosis, malnutrition, liver disease,
colon cancer. IBD represents one of the major causes of
morbidity from digestive illness, and it can be absolutely
devastating.
I want to thank our witnesses for joining us today, and
particularly Adam Carron, a very courageous young Crohn's
disease patient from my home State who is going to share his
story with us.
And I also want to recognize Bill and Shelby Modell who are
here today attending the hearing. The Modells are co-founders
of the CCFA.
I want to thank you, Mr. Chairman, for your interest in
this issue and for your leadership in convening this hearing.
As I mentioned previously, I have introduced the
Inflammatory Bowel Disease Act which would authorize an
expansion of Federal support for IBD research at the National
Institutes of Health and the Centers for Disease Control and
Prevention.
We are at an exciting time with respect to research on
these challenging diseases. A few years ago, the scientific
community discovered the first gene associated with Crohn's
disease. This is a landmark discovery, and other advancements
in the field have opened up exciting new research pathways
which have the potential to lead to better treatments and,
hopefully, 1 day soon I hope a cure.
My legislation seeks to further this momentum by
capitalizing on these promising opportunities. H.R. 290 builds
upon recommendations put forth in a concurrent resolution on
IBD, which I sponsored in the 107th Congress.
As you know, Mr. Chairman, that resolution was passed by
the Energy and Commerce Committee in September of 2002.
The Inflammatory Bowel Disease Act currently has 177
bipartisan co-sponsors in the House, including 17 members of
this distinguished subcommittee. I want to express my
appreciation to each and everyone of these members who have co-
sponsored this legislation.
Mr. Chairman, I look forward to working with you and
Ranking Member Brown who is a co-sponsor of H.R. 290, to pass
this important bill this year. H.R. 290 represents a landmark
opportunity to help improve the quality of life for IBD
patients and their families.
Thank you so much for allowing me to be here today. And I
very much look forward to hearing from our witnesses.
Mr. Bilirakis. The Chair thanks the gentlelady.
I see that the chairman of our full committee Mr. Barton
has joined us. Joe, would you like to make an opening
statement.
Chairman Barton. Thank you, Mr. Chairman. I will submit my
formal statement in the record.
I want to thank you for holding this hearing. I want to
thank Congresswoman Kelly for the effort that she has put into
this issue for many years. And hopefully we can have a good
hearing today and create a bipartisan sense of support to move
some legislation.
[The prepared statement of Hon. Joe Barton follows:]
Prepared Statement of Hon. Joe Barton, Chairman, Committee on Energy
and Commerce
Thank you, Chairman Bilirakis, for holding this hearing today.
Talking about digestive diseases is not easy. These diseases, however,
are serious, painful and dramatically impact how many Americans carry
out their daily lives. I am pleased that the Committee is starting a
dialogue about improving our current efforts to address this problem.
In preparation for this hearing, I was informed that relatively
little data exists regarding the causes, prevalence and control of
digestives disease. Although scientists have made considerable progress
in understanding the many complex diseases that affect the digestive
system, the lack of solid data limits researcher's abilities to
pinpoint the causes of these diseases and identify new treatment
options. In addition, this lack of data limits our understanding of how
many individuals are afflicted and whether these numbers are
significantly increasing.
This lack of basic data is one more example of why I am making it a
priority to reauthorize critically important public health agencies
like the National Institutes of Health (``NIH'') and the Centers for
Disease Control and Prevention (``CDC''). As part of this effort, the
Committee will explore how the CDC conducts surveys and how their data
collection activities may be improved. This review should help us
identify how the CDC can make improvements that will bring real
benefits to patients suffering from digestive diseases, in addition to
many other infectious and chronic diseases.
I am pleased that Dr. Spiegel is here to talk about how the
research programs underway at the National Institutes of Health. These
programs hold the potential to fundamentally improve our understanding
of digestive diseases. As Director of the National Institute of
Diabetes and Digestive and Kidney Diseases for the past five years, and
its scientific director for the previous 9 years, Dr. Spiegel has been
at the forefront of scientific discoveries that have already improved
patient outcomes.
By now, it is no secret to anyone that I am very committed to
making improvements at our public health agencies, and particularly at
the National Institutes of Health. Although NIH's research portfolio is
largely dedicated to basic research that transcends disease specific
research, applying this research so that it directly benefits patients
suffering from specific diseases is critical. I look forward to
learning more about the scientific opportunities NIDDK is exploring and
how we can encourage these developments by improving the organization
and structure of NIH to maximize our investments in public health.
Once again, I appreciate all of the time the witnesses have taken
to make this an informative hearing. Thank you for helping us to raise
awareness about digestive diseases so that more Americans recognize the
symptoms of these diseases and can begin treatments early.
Mr. Bilirakis. I thank the gentleman.
[Additional statement submitted for the record follows:]
Prepared Statement of Hon. Eliot Engel, a Representative in Congress
from the State of New York
Mr. Chairman, thank you for having this hearing today. Digestive
diseases affect millions of Americans and I believe it is highly
appropriate that our Committee examine the issues and the potential for
moving research forward.
Mr. Chairman, I am pleased to welcome our witnesses. I appreciate
all of their time and especially the testimony of Adam Carron from the
Crohn's and Colitis Foundation of America. I appreciate your courage in
coming before the Committee this morning to talk about your experienced
with IBD. I also welcome Rodger DeRose from the Crohn's and Colitis
Foundation. Both are from my home state of New York, and they have been
instrumental in raising awareness about these diseases and helping
patients in New York City and across the country. I am pleased to be a
co-sponsor of H.R. 290, legislation focused on Crohn's disease and
ulcerative colitis. This legislation has the support of 175 bipartisan
members of the House. I look forward to working with the Chairman and
Ranking Member to advance this important legislation in the Committee
this year.
I am also interested in hearing what work the NIH is pursuing with
regards to Irritable Bowel Syndrome (IBS). Obviously IBS doesn't get
nearly the attention of IBD because it is a less severe condition and
not life threatening but it still terribly disrupts the lives of its
victims and we need to focus on this condition as well. I look forward
to hearing from Dr. Spiegel about NIH's research efforts and a
strategic plan regarding IBS.
Mr. Chairman, I thank you for your efforts to examine digestive
disease and look forward to working with you on this important issue.
Mr. Bilirakis. All right. Let us go right into the
testimony part of the witnesses. I have already introduced
them.
Dr. Spiegel is the Director of National Institute of
Diabetes and Digestive and Kidney Diseases here in Bethesda.
Mr. DeRose is President and CEO of the Crohn's and Colitis
Foundation of America. Adam Carron is a young man who
unfortunately has been inflicted with this disease, but I think
that is probably God's way of saying to Adam hey this is your
opportunity to help an awful lot of people around the world by
virtue of your personal experience. And Dr. Peura is here on
behalf of the Digestive Disease National Coalition.
Your written statements are already a part of the record.
And I would hope, I am going to set this at 5 minutes. I will
not cut you off, but I would hope you would stay as close to it
as you can. But hopefully you would sort of supplement and
compliment your written statement.
Dr. Spiegel, let us start off with you, sir.
STATEMENTS OF ALLEN M. SPIEGEL, DIRECTOR, NATIONAL INSTITUTE OF
DIABETES AND DIGESTIVE AND KIDNEY DISEASES; RODGER DeROSE,
PRESIDENT AND CEO, CROHN'S AND COLITIS FOUNDATION OF AMERICA;
ADAM CARRON; AND DAVID PEURA, DIGESTIVE DISEASE NATIONAL
COALITION
Mr. Spiegel. Thank you very much, Mr. Chairman and members
of the committee. I am please to testify today regarding NIH
efforts to combat digestive diseases.
I am accompanied today by Dr. Stephen James, who is the
newly appointed Director of the Institute's Division of
Digestive Diseases and Nutrition. He's an expert on digestive
disease research, particularly immunologically mediated
diseases including the inflammatory bowel diseases.
I have provided the written statement with a detailed
account of the public health burden of digestive diseases, the
vigorous research efforts NIH has underway in this area and
future research directions based on our planning. I would like
to just give you a brief overview this morning.
Digestive diseases research encompasses many serious,
potentially life threatening illnesses. Examples, hepatitis,
gastrointestinal cancer as well as highly prevalent diseases
such as acute gastroenteritis, gastroesophageal reflux, the
cause of heartburn, and irritable bowel syndrome. While these
generally are not fatal, they cause significant morbidity.
In fiscal year 2003 the NIH invested nearly $1.2 billion in
research on digestive diseases. The National Institute of
Diabetes and Digestive and Kidney Disease, which I head, the
National Cancer Institute and the National Institute of Allergy
and Infectious Diseases accounted for 34, 32 and 17 percent of
support, respectively.
During the period of the doubling of the NIH budget for
which, by the way, we're extraordinarily appreciative of the
bipartisan efforts of this Congress, NIDDK was able to fund a
significant number of large scale digestive disease research
initiatives. We were able to fund four new digestive disease
development centers, a women's health center focusing on
irritable bowel syndrome and to expand the number of digestive
disease research centers from 12 to 16, including our newest
one at the University of Virginia where another panel member,
Dr. Peura, is the associate chief of the gastroenterology
division.
There are active collaborations in digestive disease
research among many NIH components. Just one example, NIDDK and
NCI have joint efforts on Barrett's esophagus, which can be a
precursor to cancer of the esophagus. Also, we collaborate on
the diagnosis and treatment and prevention of liver cancer,
which may be a consequence of infection with the hepatitis
virus.
The NIDDK plays an important role in disseminating
information on digestive disease through the National Digestive
Diseases Information Clearinghouse. And this speaks to the
chairman's point about the need not only to do the research,
but to get the information out to the American public.
The clearinghouse develops and distributes health
information for patients, the public and healthcare providers
to improve understanding of digestive diseases. Just last week
the NIH sponsored a consensus development conference on celiac
disease, an immune mediated disorder that primarily affects the
digestive tract. This disease affects about 1 percent of the
U.S. population, but is currently recognized in only about one-
tenth of patients with current medical practice. In addition to
providing useful guidance to the NIH, we are hopeful this
conference will raise awareness of the disease among medical
practitioners and the public in order to promote early,
accurate diagnosis and treatment.
As a major example of NIH digestive diseases research, I
would like to give the committee a brief description of
research advances in the inflammatory bowel diseases,
ulcerative colitis and Crohn's disease. Many patients are
diagnosed in childhood or in their teens and must cope for the
rest of their lives with problems that include intestinal
inflammation, abdominal pain, fever, diarrhea and rectal
bleeding. We will hear momentarily from Mr. Carron whose
testimony, I am sure, will be more eloquent than anything I
could say about the impact of these inflammatory bowel
diseases.
Now for decades there were no truly effective medications.
Surgical removal of the affected parts of the intestine was
often necessary, particularly in ulcerative colitis which can
lead to colon cancer. Investigator initiated basic research on
the immune system, however, began to illuminate the fundamental
mechanisms responsible for intestinal inflammation. These
discoveries set the stage for an ambitious long range plan for
IBD research with the goals of improving lives with more
effective treatment and ultimately, prevention. The plan,
importantly, was formulated with external input from patient
groups such as the Crohn's and Colitis Foundation of America
and investigative groups such as the American
Gastroenterological Association, both valued partners of NIDDK.
Through NIH research efforts significant improvements in
therapy for Crohn's disease have resulted, notably the
development of infliximab, a drug that targets an inflammation
causing protein. The FDA's approval of infliximab was an
important step forward in treating Crohn's disease.
Then as Congresswoman Kelly has alluded to, a major
discovery occurred in 2001 when investigators announced the
discovery of a gene that confers susceptibility to Crohn's
disease. It is fine to find a new gene, but you need to
actually then build on that to do something for patients, that
is really the important part; and we have. The role of this
particular gene illuminated that abnormalities in the innate
immune system, the first line of defense against foreign
invaders, was an important potential cause of Crohn's disease.
This was translated into a clinical trial demonstrating the
potential benefit of a new treatment which boosts the function
of the innate immune system. Because other genes conferring
susceptibility to inflammatory bowel disease remain to be
discovered and could provide similar insights, the NIDDK
established a new multicenter genetics consortium to speed this
research.
In another example, and I think a particularly important
one in terms of the crosscutting nature of research, NIDDK
investigators demonstrated that the drug Rosiglitazone,
currently used to treat diabetes, has anti-inflammatory effects
in an animal model of IBD. Subsequently, an NIDDK sponsored
multicenter clinical trial of this drug for treatment of
ulcerative colitis has been initiated and is currently in
progress.
There are several promising agents in the pipeline and I
hope the committee sees, as these highlights show, that
progress in IBD research reflects a convergence of public
health need, stakeholder input, scientific opportunity and the
critical peer review system which assures that only the most
meritorious proposals submitted to the NIH are funded.
On a broader level the NIH is now embarking on a new
planning process for digestive diseases generally under the
auspices of the statutory Digestive Diseases Interagency
Coordinating Committee chaired by Dr. James. The first step in
that process is the development of a liver disease research
action plan, a commitment that I made to the chairman back in
September of 2002. This is being done in consultation with
external scientific and lay experts. We will be following a
similar planning process for other specific digestive diseases.
We believe this planning effort will produce useful guideposts
for prioritization in NIH program development and importantly,
will help synergize cross-cutting research efforts across the
NIH.
Finally, let me mention that on March 9th Dr. James and I
were pleased to present an overview of the digestive disease
research program, recent advances and future plans to the newly
created Congressional Digestive Disease Caucus. The caucus was
founded through the leadership of Congresswoman Sue Kelly, and
we are really appreciative of her commitment as Chair of the
caucus to increase awareness of the burden of digestive
diseases and to encourage research in this important area.
Mr. Chairman and members of the committee, I hope these few
examples convey the firm commitment of the NIH to combatting
the many digestive diseases within its research mission.
Through research we seek to relieve the burden these chronic,
debilitating, frustrating diseases place on individuals,
families and the nation.
I appreciate the opportunity to address the committee on
behalf of the NIH and the NIDDK, and we would be pleased to
respond to any questions you may have.
[The prepared statement of Allen M. Spiegel follows:]
Prepared Statement of Allen M. Spiegel, Director, National Institute of
Diabetes and Digestive and Kidney Diseases, National Institutes of
Health, U.S. Department of Health and Human Services
Mr. Chairman and Members of the Committee: I am Allen Spiegel,
Director of the National Institute of Diabetes and Digestive and Kidney
Diseases (NIDDK), the Institute that has lead responsibility for
digestive diseases research at the National Institutes of Health (NIH
of the Department of Health and Human Services. I am pleased to testify
today regarding NIH efforts to combat digestive diseases. Through basic
and clinical research studies, we can gain greater insights into the
causes of these diseases, find more effective treatments, and develop
prevention strategies. I am accompanied today by Dr. Stephen James, the
newly appointed Director of the Institute's Division of Digestive
Diseases and Nutrition. Dr. James is an expert on digestive disease
research, particularly immunologically mediated diseases, including
inflammatory bowel diseases.
In my testimony today, I will give you a brief overview of the
public health burden of digestive diseases, the vigorous research
efforts NIH has under way in this area, highlighting Crohn's disease
research as an illustrative example, and closing with future directions
based on our planning for research in digestive diseases. BURDEN OF
DIGESTIVE DISEASES
The digestive system is critically important to human health and
well being. This complex system includes the pharynx, esophagus,
stomach, liver, biliary tract, pancreas, small and large intestines,
and anorectum. Thus, digestive diseases research encompasses many
serious and potentially life-threatening illnesses, such as cirrhosis
of the liver, inflammatory bowel diseases, hepatitis, gastrointestinal
cancer, ulcers, and gallstones. This constellation of diseases also
includes highly prevalent diseases such as acute gastroenteritis,
gastroesophageal reflux disease (the cause of heartburn), and irritable
bowel syndrome that, while generally not fatal, cause significant
morbidity.
Digestive diseases and their associated long-term complications
have significant social and economic consequences for the Nation.
According to a report published in 2002, the estimated annual total
cost for digestive diseases, in 1998 dollars, was $85.5 billion
(Sandler RS, Everhart JE, Donowitz M, Adams E, Cronin K, Goodman C,
Gemmen E, Shah S, Avdic A, Rubin R. The burden of selected digestive
diseases in the United States. Gastroenterology. 2002 May; 122(5):1500-
11).
Digestive diseases rank second among all causes of disability due
to illness in the United States. They result in an estimated 200,000
absences from work each day with a mean time lost of nine days. More
than two million Americans are impaired to some degree by digestive
diseases, limiting an estimated 1.2 million people in the type of
occupation they can seek. Approximately 140,000 veterans receive
payments for service-related disabilities due to digestive diseases.
The chronic nature of digestive diseases results in approximately
11 percent of all admissions to general hospitals in the United States
and in 15 percent of all surgical procedures performed in this country.
Approximately 200,000 deaths annually are caused by digestive diseases,
including cirrhosis and other liver diseases, cancer of the digestive
system, gallbladder disease, ulcers, and pancreatitis. Digestive
diseases also complicate the treatment of other life-threatening
conditions, such as cardiovascular disease.
In fiscal year 2003, the NIH invested nearly $1.1 billion in
research on digestive diseases. The NIDDK, the National Cancer
Institute (NCI), and National Institute of Allergy and Infectious
Diseases (NIAID) accounted for 34 percent, 32 percent, and 17 percent
of this support, respectively. Research mechanisms include regular
research grants, cooperative clinical trials, epidemiologic studies and
data systems, and cooperative consortia. In addition, during the period
of the doubling of the NIH budget, we were able to expand the number of
NIDDK Digestive Disease Research Centers from 12 to 16, as well as to
add four new digestive disease Development Centers, plus a Women's
Health Center (with the NIH Office of Research on Women's Health)
focusing on irritable bowel syndrome. We are also vigorously supporting
physician-scientists in digestive diseases through our research
training and career development awards and the loan repayment program.
A statutory Digestive Diseases Interagency Coordinating Committee
serves to coalesce and synergize the efforts of the many NIH Institutes
and Centers that support research in this field, as well as the efforts
of other Federal agencies. Intersecting research and active
collaboration are found among many NIH components. For example, NIDDK's
research on the development of islet cells of the pancreas complements
the NCI's work on the cellular origins of pancreatic cancer. Similarly,
NIDDK and NCI have joint efforts on both Barrett's esophagus, which can
be a precursor to cancer of the esophagus, and on the diagnosis,
treatment, and prevention of liver cancer, which may be a consequence
of infection with hepatitis virus. Hepatitis is a shared research focus
of the NIDDK, NIAID, the National Institute on Alcohol Abuse and
Alcoholism, and the National Institute on Drug Abuse. The NIDDK and the
National Center for Complementary and Alternative Medicine are working
together to test silymarin, or milk thistle, in treatment of liver
disease. These are just a few examples of the many ongoing
collaborative endeavors at NIH in digestive diseases research.
The NIDDK also plays an important role in disseminating information
on digestive diseases through the National Digestive Diseases
Information Clearinghouse (NDDIC). The Clearinghouse develops and
distributes health information for patients, the public, and health
care providers to improve understanding of digestive diseases, such as
Crohn's disease (http://digestive.niddk.nih.gov/ddiseases/pubs/crohns/
index.htm). The Clearinghouse is available via the web
(www.digestive.niddk.nih.gov), a toll-free phone line (1-800-891-5389),
e-mail ([email protected]), mail (NDDIC, 1 Information Way,
Bethesda, MD 20892-2570), and fax (301-907-8906). Each year, the
Clearinghouse meets with representatives of professional and patient-
advocacy groups to share information and seek feedback. The NDDIC's
most recent national meeting was held on June 10, 2004, during which
Michael Dolan reported on activities at the Crohn's and Colitis
Foundation of America.
INFLAMMATORY BOWEL DISEASES: NEW DISCOVERIES AND PROMISE FOR THE FUTURE
As a major example of NIH digestive diseases research, I would like
to give the committee a brief description of the paths that have been
taken to realize research advances in the inflammatory bowel diseases
(IBD), ulcerative colitis and Crohn's disease. Ulcerative colitis is
characterized by inflammation and ulceration of the inner surface of
the large intestine. Crohn's disease may involve any portion of the
gastrointestinal tract, but most commonly affects the lower portion of
the small intestine. The lesions of Crohn's disease may penetrate
through the bowel wall and lead to the formation of fistulas, which are
ulcerating lesions that tunnel through the intestines of patients.
The inflammatory bowel diseases are incurable, chronic, and
debilitating. They affect an estimated 1 million Americans. Many
patients are diagnosed in their teens and twenties and must cope for
the rest of their lives with problems that include intestinal
inflammation, abdominal pain, fever, diarrhea, and rectal bleeding. In
children, symptoms can progress to malnutrition and growth retardation.
These problems dramatically reduce quality-of-life and require
lifelong, expensive medical care. For decades, there were no truly
effective medications, so that surgical removal of the affected parts
of the intestine was often necessary, particularly in ulcerative
colitis, which can lead to colon cancer.
Investigator-initiated basic research on the immune system,
however, began to illuminate the fundamental mechanisms responsible for
intestinal inflammation, leading to important therapeutic advances,
particularly in Crohn's disease. Mice in which certain key genes of the
immune system had been knocked out unexpectedly developed inflammatory
bowel disease mimicking Crohn's disease. Importantly, bowel disease did
not develop in mice raised under ``germ-free'' conditions in which the
bacteria normally residing in the bowel are absent. Thus, the immune
gene knockout alone is insufficient to cause disease, but combines with
the normal gut bacteria in provoking a self-destructive immune
response.
These discoveries set the stage for an ambitious long-range plan
for inflammatory bowel disease research with the goals of providing
more effective treatment and, ultimately, prevention. The NIDDK has
pursued the plan's aims to: (1) emphasize basic research on
interactions between intestinal cells and bacteria; (2) augment the
pool of researchers and foster interdisciplinary research; (3)
establish and enhance appropriate biological resources and data
collections; and (4) develop therapeutic applications and preventive
approaches as basic research progresses.
This plan was formulated with external input from patient groups,
such as the Crohn's and Colitis Foundation of America, and investigator
groups, such as the American Gastroenterological Association. Dr. James
has worked closely with these advocacy groups to maximize efforts
toward accomplishing common research goals. The plan was updated at a
meeting of the NIDDK-led Digestive Diseases Interagency Coordinating
Committee in April 2003. In implementing the plan, the NIDDK has
deployed the full range of available mechanisms, including a robust
portfolio of investigator-initiated grants and pilot-and-feasibility
studies, research training grants, large program project grants, and
four Digestive Diseases Research Centers that focus on inflammatory
bowel disease.
Through NIH research efforts, significant improvements in therapy
for Crohn's disease have resulted, most notably the development of
infliximab, a drug that targets an inflammation-causing protein whose
role was illuminated by the mouse gene knockout experiments. The Food
and Drug Administration's (FDA) approval of infliximab was an important
step forward in treating Crohn's disease. A report in the New England
Journal of Medicine earlier this year showed that it is particularly
effective in healing the fistulas. An enormous new scientific
opportunity emerged in 2001 when investigators announced the
unprecedented discovery of a gene that confers susceptibility to
Crohn's disease. This discovery represents an important payoff of the
Human Genome Project. It is also a credit to the strong foundation laid
by previous NIDDK research efforts--including an emphasis on targeted,
interdisciplinary collaborations among researchers from different
fields. Finding this gene illuminated the potential role of
abnormalities of the innate immune system--the first line of defense
against ``foreign invaders''--as a cause of Crohn's disease. This
insight was then translated into a clinical trial demonstrating the
potential benefit of a new treatment, GM-CSF, a natural protein in the
body which boosts the function of the innate immune system. Because
other genes conferring susceptibility to inflammatory bowel disease
remain to be discovered, the NIDDK established a new multi-center
Genetics Consortium to speed this search. The pace of discovery in
genetics of IBD is accelerating, as evidenced by the publication in
April of the identification of two new candidate genes involved in
Crohn's disease.
Most recently, results from a multicenter clinical trial were
presented at the May 2004 meeting of the digestive diseases
professional organizations. Researchers have shown that there is a
benefit for Crohn's patients in the use of a monoclonal antibody
targeting the cytokine IL-12. This work emanates from the intramural
research program of the NIAID.
Other clinical insights are expected to emerge from an ongoing
NIDDK-funded multicenter clinical trial to investigate whether the
dosing of the standard, now generic, immunosuppressive drug
azathioprine can be improved. Researchers are studying new testing
methods for the metabolizing enzyme and toxic metabolites of the drug.
This trial is an example of the vital role NIH can play in conducting
clinical trials of a treatment when there is no longer any incentive
for commercial interest in such research.
Other NIDDK initiatives include the detailed study of adult stem
cells of the intestine that may ultimately lead to therapies
stimulating gut regeneration. We are vigorously pursuing these research
areas as we evaluate and monitor progress in attaining our goals. While
this example illustrates how investments in investigator-initiated
basic research lead to discoveries that improve the outcomes of
patients with Crohn's disease, we recognize that we must take steps to
accelerate the translation of basic science discoveries into patient
benefits. For this reason, NIH and NIDDK are taking steps to bolster
translational research.
INFLAMMATORY BOWEL DISEASES: TRANSLATIONAL RESEARCH OPPORTUNITIES
The NIH is strongly committed to spurring translational research in
both Crohn's disease and ulcerative colitis. By translational research,
we mean research to speed the movement of laboratory discoveries into
research that holds promise of direct clinical benefits for patients--
also called ``bench-to-bedside'' research. NIH Director Elias Zerhouni,
M.D., has stressed the importance of this type of research in his
initiative to develop a Roadmap for medical research, and the many
Institutes and Centers of the NIH are also emphasizing translation
research in their respective programs. At the NIDDK, for example, we
have recently completed an assessment of drugs in the pipeline for
several diseases within our mission, including the inflammatory bowel
diseases--Crohn's disease and ulcerative colitis. One good example of
translational research involves the drug rosiglitazone, which is used
to treat diabetes. NIDDK-funded investigators demonstrated that this
drug has anti-inflammatory effects in an animal model of IBD, and
subsequently, a NIDDK-sponsored multi-center clinical trial of this
drug for treatment of ulcerative colitis has been initiated and is
currently in progress. There are approximately 10 new drugs under
development for one or both of these diseases, as well as many new
studies of existing agents. The novel therapies have emerged from a
foundation of basic research supported by the public sector, with drug
development steps pursued by the private sector. We are encouraged when
industry builds upon NIH-funded basic research discoveries because such
activity offers promise of new and more effective treatments for IBD.
We have identified roadblocks to translational research in IBD and
steps that can be taken to address them, such as the development of
surrogate markers of disease activity and better diagnostic tests. It
is also essential to maximize the research investment in animal models
of IBD, which can continue to provide insights into the underpinnings
of the disease, and also serve as a source of potential genetic
discoveries and a means of testing emerging new therapies. Research
progress is often hampered by the difficulty of obtaining access to
human samples. To overcome this barrier, NIDDK has recently initiated a
repository that will collect and make available to investigators
various types of human samples, including blood, biopsies, genetic
material, and datasets. Another barrier to clinical research concerns
the great complexity of modern clinical trial designs. To facilitate
testing of additional new treatments under development, the NIDDK
convened a meeting in January 2003 that included representatives from
the FDA, industry, and the investigative community, to seek
improvements in the design of clinical trials, with emphasis on
improving trial endpoints. We will continue to foster such proactive
partnerships with the FDA and industry, and also to pursue clinical
studies in needed areas that industry does not have a commercial
incentive to explore.
As these highlights show, progress in IBD research reflects a
convergence of public health need, scientific opportunity, stakeholder
input, and the merit of research proposals submitted to the NIH for
funding. While many strides have been made, we still recognize that our
currently available therapies have many drawbacks and may not provide
the adequate symptom relief that patients need. At the same time,
however, we are encouraged by the advances being made through research
and are committed to accelerating the pace of discovery and
translation.
To this end, NIDDK is fostering more cross-cutting initiatives,
including emphasis on harnessing powerful new technologies in genomics,
proteomics, and molecular imaging to address long-standing problems.
Availability of a non-invasive imaging method to assess liver scarring
or fibrosis, for example, would transform the clinical management of
many liver diseases which now must rely on invasive biopsies. Such
cross-cutting initiatives have broad application not only to digestive
diseases, but also to a wide array of other diseases within the NIH
research mission. By pursuing such endeavors, we can help to maximize
NIH research investments by promoting their greatest yield and
application.
ENHANCING DIGESTIVE DISEASES RESEARCH
In building the digestive diseases research portfolio, we recognize
the importance of input from the scientific and lay community external
to the NIH. I would like to provide just a few examples.
Stakeholder input is an important dimension of our planning and
program development processes. As noted previously, our joint planning
efforts with the Crohn's and Colitis Foundation of America have been
very productive. Another example of input that guides NIH program
development can be found in the insights and recommendations we obtain
from a wide range of conferences and workshops. For example, in
digestive diseases, the NIH has sponsored critically important
Consensus Development Conferences on hepatitis C, and we recently
submitted to the Congress a report on our implementation of the
recommendations we received. Just last week, June 28-30, 2004, the NIH
sponsored a Consensus Development Conference on celiac disease, an
immune-mediated disorder that primarily affects the digestive tract.
This disease affects about 1 percent of the U.S. population but is
recognized in only about one-tenth of patients using current medical
practice. In addition to providing useful guidance to the NIH, we are
hopeful that this conference will raise awareness of the disease among
medical practitioners and the public in order to promote early,
accurate diagnosis and treatment.
On a broader level, the NIH is now embarking on a new planning
process for digestive diseases generally, under the auspices of the
Digestive Diseases Interagency Coordinating Committee chaired by Dr.
James. The first step in that process is the development of a Liver
Disease Research Action Plan, in consultation with external scientific
and lay experts. An open meeting provided significant input from
representatives of professional organizations, patients, and the
public. Six draft chapters of the Plan have already been posted on the
NIDDK website for additional public comment, and remaining chapters
will be posted as they are completed by the Committee. We will be
following a similar planning process for other specific digestive
diseases, and we believe that this planning effort will produce useful
guideposts for prioritization in NIH program development, and will help
synergize cross-cutting research efforts across the NIH.
Finally, I also want to mention that on March 9, Dr. James and I
were pleased to present an overview of the NIDDK digestive disease
research program, recent advances, and future plans to the newly-
created Congressional Digestive Disease Caucus. The Caucus was founded
through the leadership of Congresswoman Sue Kelly. We are most
appreciative of her commitment, as Chair of the Caucus, to increase
awareness of the burden of digestive diseases and to encourage research
in this important area.
Mr. Chairman and Members of the Committee, I hope that these few
examples convey the firm commitment of the NIH to combating the many
digestive diseases within its research mission. Through research, we
seek to relieve the burden these chronic, debilitating, frustrating
diseases place on individuals, families, and the Nation. I appreciate
the opportunity to address the Committee on behalf of the NIH and the
NIDDK, and would be pleased to respond to any questions you may have.
Mr. Bilirakis. Thank you very much, Dr. Spiegel.
Mr. DeRose, you are on, sir.
STATEMENT OF RODGER DeROSE
Mr. DeRose. Well, thank you, Mr. Chairman. I appreciate the
opportunity to testify on behalf of the patients across the
country.
I think you all have background now in terms of the
complications of this disease, so I am not going to be
repetitive in that. I think when you hear Adam's story, you
will understand the dilemma that so many of these patients
face; that you look at a young man like this that looks healthy
and at the same time you know that on the inside they are not
healthy. And that is the deceiving part about this disease. And
we find this as we go across the country talking to patients
that we do not get our due respect because individuals see our
patients and do not recognize how debilitating this disease can
be.
I just wanted to give you some background in terms of our
foundation. We were founded in 1967. And, actually, two of our
co-founders are here today, Mr. Chairman. I want to recognize
them, Bill and Shelby Modell from New York. And, you know, they
started this organization with the Rosenthals in 1967 because
they had a teenager that had the disease. And a few years ago
they lost their son, Michael, to not complications to Crohn's
but the fact that his immune system was compromised so
dramatically that other diseases set in. And they lost him.
They have continued to be 100 percent committed to this
organization, day in and day out.
From the time that we have started we have expanded to 42
chapters around the country. We have a mission of research,
education and support. To date, we have raised nearly $300
million, $100 million of that has gone directly into research.
Because we have such a high efficiency rate as an organization,
the majority of the balance of that $200 million has gone into
education and support to the patient community.
And I think it is fair to say, Mr. Chairman, that the
organization in its 30 plus years history has an impact on
every major aspect of this disease in terms of improving the
quality of life of the patient community in terms of: New
therapies that have been introduced because of the basic
science that CCFA has supported; safer alternatives to steroids
that were available, only available 25 years ago; advanced
surgical techniques that have given patients their life back;
improved diagnostic tools that have allowed us to detect the
disease much earlier; in the area of early warning signs in
terms of dysplasia and cancer, and; also in the area of
nutritional therapies because many of our patients are
malnourished. They cannot get the calories into their system
because they're going to the bathroom so often and flushing
this through their system. And importantly, education programs.
In the area of education while we run these education
programs throughout the country and over 300 support groups
around the country each year, we also reach out to the children
with this disease in terms of running camps. We have 14 camps
across the country that help children cope with the physical as
well as emotional battles that they deal with with this
disease. It is staffed by volunteer doctors, volunteer nurses
and camp counselors 7 by 24.
So it is a community that is really based on volunteerism
and has a dramatic impact in the advances that are taking place
in this disease state.
I want to say one thing about the number of patients that I
have talked about. We conservatively estimate that it is a
million Americans, but we happen to believe that it is probably
much higher than that. Anecdotally as we travel the country, as
we get phone calls from patients and we talk to the
professional community, it is our understanding that we are
probably underestimating the size and the impact of this
disease across our country. And as a result of that, the
foundation 2 years actually went to the Center for Disease and
Control and Prevention and we actually went to them and ask
them to submit a grant request to CCFA so that we could start
an epidemiology program with them, an epidemiology study to
better understand the prevalence of this disease. So we funded
it to the tune of three-quarters of a million dollars for those
2 years.
And our scientific advisory committee has been working very
closely with the CDC, but despite repeated encouragement from
your colleagues, Mr. Chairman, on the Appropriations Committee
the CDC has yet to provide any financial support for this
project. And now that the study has been initiated by funding
from a patient group to a government agency, we are hoping that
the CDC will contribute to the completion of this major study.
And, of course, that is one of the aspects of the IBD bill.
With respect to research, I have to tell you that it is a
real privilege for us to work in partnership with the NIH
within NIDDK and in particular with Dr. James as well as Dr.
Allen Spiegel. Dr. James actually sits as an ad hoc member, a
nonvoting member of our national scientific advisory committee.
And so we have a very close working relationship with them, and
we exchange intellectual knowledge as we try to advance the
state of this disease on behalf of the patient community.
I think one good example of that is the benefits that this
approach has led to in terms of the discovery of the very first
gene that was talked about were CCFA funded that and then the
NIH came in and continued to invest the furtherance of
understanding, the impact of that gene as well as other genes
that may lead to getting us closer to the cure.
We are very proud of the support that we have had and that
we have played with the NIH. If you talk to some of the
investigators, they would say that there is probably a 20 times
multiplier of fact in terms of from the time that they first
get their CCFA grant from us to the time that they meet the
midpoint of their career in terms of the additional funding
that they get from the NIH. So you can see that there is an
infusion of knowledge that is taking place here from the
preliminary data that CCFA is providing with the research that
the NIH is picking up and continuing.
So I am confident that working together that we can make
dramatic strides here.
Mr. Chairman, I do want to thank Congresswoman Sue Kelly
for introducing the bill as well as Congressman Jessie Jackson,
Jr. And I thank you for taking the time out to hear our
testimonies today and everything that you are doing on behalf
of the patient community to advance the quality of life for our
patients.
Thank you very much. And I would be happy to answer
questions whenever you are ready.
[The prepared statement of Rodger DeRose follows:]
Prepared Statement of Rodger DeRose, President and Chief Executive
Officer, Crohn's and Colitis Foundation of America
Mr. Chairman and members of the subcommittee, thank you for the
opportunity to testify today. I am Rodger DeRose, President and Chief
Executive Officer of the Crohn's and Colitis Foundation of America.
CCFA is the nation's oldest and largest organization dedicated to
finding a cure for inflammatory bowel disease. IBD (which includes both
Crohn's disease and ulcerative colitis), is a chronic disorder of the
gastrointestinal tract which afflicts conservatively 1 million
Americans, 100,000 or 10% of whom are children under the age of 18. IBD
can cause severe diarrhea, abdominal pain, fever, and rectal bleeding.
Complications related to the disease include; arthritis, osteoporosis,
anemia, liver disease, and colon cancer. IBD represents a major cause
of morbidity from digestive illness, and although it is not usually
fatal due to the major advances that have occurred in the last 30
years, IBD can be devastating. We do not know its cause, and there is
no medical cure.
Founded in 1967, CCFA is headquartered in New York City and has 42
chapters that provide education and support services to patients in all
50 states. Since its beginning, CCFA has generated $300 million in
revenues--$100 million of which has been directly invested in basic and
clinical research related to IBD. Because CCFA prides itself on being
an efficient organization, with low administrative costs, a large
percentage of the remaining $200 million has been reinvested in patient
education and support programs.
Through CCFA's leadership, our organization has impacted every
major advancement in Crohn's and colitis over the past 30 years
including: new therapies and safer alternatives to steroids, advanced
surgical techniques, improved diagnostic methods to detect the disease
earlier, early warning of dysplasia for the treatment of colorectal
cancer in patients, nutritional therapies for struggling patients who
can't get calories into their malnourished bodies, and education
programs for both the patient and professional community.
In the area of education, CCFA plays a leadership role in patient
based training through our 42 chapters and the 300 support groups that
we offer to patients each year. Additionally, we reach out to children
with IBD by sponsoring 14 camps throughout the US to help kids deal
with the physical and emotional elements of this disease. We staff the
camps with volunteer doctors, nurses and camp counselors that are
available 24 hours a day, 7 days a week to meet the needs of the kids.
As I mentioned earlier, it is conservatively estimated that 1
million Americans suffer from either Crohn's disease or ulcerative
colitis. CCFA and other leaders within the digestive disease community
believe, however, that the actual number of patients is significantly
higher. As a result, the Foundation has provided the Centers for
Disease Control and Prevention with $750,000 over the past two years to
initiate a national IBD epidemiology study to determine the true
prevalence of the disease.
Gaining a better understanding of the number of patients afflicted
with IBD, and their demographic characteristics, will give us
invaluable clues as to the role that environmental factors play in the
development and progression of the disease.
CCFA's National Scientific Advisory Committee has developed a
strong working relationship with the CDC on this important study.
However, Mr. Chairman, despite repeated encouragement from your
colleagues on the Appropriations Committee, CDC has yet to provide any
financial support for the project. Now that the study has been
initiated by an investment from the patient community, we are hopeful
that CDC will contribute to the continuation and completion of this
major study. Mr. Chairman, we would welcome your support of this
important initiative as we move forward.
With respect to research, the IBD community is encouraged by the
significant progress that has been made in recent years. We are
extremely grateful for the leadership of Dr. Spiegel, and his colleague
Dr. Stephen James, at the NIDDK. They are strong partners in our
continuing effort to combat this disease.
However, we all agree that much more needs to be done. To that end,
CCFA's scientific leaders have developed a forward-thinking research
agenda entitled, ``Challenges in Inflammatory Bowel Disease'' that
outlines the many exciting opportunities that currently exist in the
field. Next to the NIH, CCFA is the leading source of funding for IBD
research. CCFA plays a critical role in providing ``seed funding'' to
researchers interested in IBD. This support helps investigators gather
preliminary findings which, in turn, enables them to pursue advanced
IBD research projects through the NIH. A good example of the benefits
of this approach is the research that led to the discovery of the first
gene associated with Crohn's disease in 2001. Preliminary support for
this historic research was provided by CCFA and enhanced by the NIDDK
and other funding sources in subsequent years.
We are proud of the enormous impact that support from CCFA has had
on Crohn's and colitis research. Some investigators estimate that there
is a 20 factor multiplier effect from the time a CCFA funded
investigator receives their first CCFA grant to the peak of their
career. In fact, more than 80% of CCFA-sponsored researchers have
obtained subsequent funding from the NIH for further IBD research.
Mr. Chairman, I am confident that working together, with additional
resources, we will develop better treatments and eventually a cure for
these diseases. In 5 years, I'm hopeful that we will have approximately
5 biologic treatments available to target the disease. Today we have 1
FDA approved biologic with several pursuing or finishing stage 3
trials. In 10 years, I am hopeful that through our genetic
understanding of the disease that we will have genetic treatments to
alter the course of the disease. We are at an unprecedented time of
optimism with respect to research on IBD. Opportunities for advancement
and real progress have never been greater. An additional investment in
this area has the potential to yield significant and tangible results
for patients.
Mr. Chairman, I would like to take this opportunity to thank
Congresswoman Sue Kelly and Congressman Jesse Jackson, Jr. for their
leadership in introducing the ``Inflammatory Bowel Disease Act'' in the
House. This landmark legislation, which addresses IBD research at the
NIH and CDC, as well as other issues that are important to our
community, has over 175 bipartisan co-sponsors, including 17 members of
this distinguished subcommittee. On behalf of CCFA and the entire IBD
community, I want to express our sincere appreciation for all of the
support that this important legislation has received. Our patients and
family members across the nation encourage the subcommittee to pass
legislation focused on inflammatory bowel disease this year.
The chances are high that most of the subcommittee members here
know someone with this disease: a co-worker, a family member, friend,
acquaintance. What you may not know is the symptoms that they live with
daily because most look normal outwardly but they are anything but
normal on the inside.
We need your help in bringing Crohn's and colitis out of the closet
so patients can get the support they need in seeking the cure through
the NIH and the CCFA. You can be sure that the CCFA will do its part to
end the disease.
In closing Mr. Chairman, I want to thank you for your interest in
this disease and for convening this important hearing today. Our Long
Island chapter was honored that you took time out of your busy schedule
to join us for our annual dinner in May. Our Long Island Chapter
President, Jamie Pappas and his family, send you their best. We are
very grateful for your leadership and look forward to working with you
to help improve the quality of life for IBD patients and their
families.
I would be pleased to respond to any questions that you may have.
Mr. Bilirakis. Unfortunate news that we have 3 votes on the
floor.
Let us go into Adam.
STATEMENT OF ADAM CARRON
Mr. Carron. Mr. Chairman and members of this committee,
thank you for the opportunity to testify today.
I am Adam Carron, an 18 year old ulcerative colitis patient
from Long Island, New York and I appreciate the opportunity to
share my story about living with inflammatory bowel disease
with you.
My experience with IBD began at age 9. It started with
bloody stools and intense abdominal pian, and my parents'
nightmare came true when my doctor told us all that I was
probably suffering from ulcerative colitis; the same disease
that lead to the death of my uncle at age 42 and the illness my
mother was struggling with.
Before I could even pronounce ``inflammatory bowel
disease,'' let alone know what it was, I knew I had something
bad and was in serious trouble. I even feared that I was going
to die.
I remember the insecurities affecting my everyday life;
backing out of sleeping at friends' houses and going to camp;
even being at school needing permission to use the bathroom
frequently during the day fearful of what might happen if my
teachers said no.
I was given the responsibility to remember to take my
medicine every day, both orally and anally, just so that I
would not have stomach cramps and blood in my stools. I
resented it because in a time when all I wanted was to fit in,
none of my friends had the same issues, and therefore I did not
always follow through.
But at age 11 I got very sick. The medications were no
longer able to control my symptoms. I was bleeding profusely
and had severe pain. I could not stop vomiting. I was forced to
go into the hospital and for several weeks I lay there being
given massive doses of steroids. I will never forget having to
miss trick or treating with my friends and then not being able
to eat the Halloween candy my sister had so graciously gathered
for me because I could not eat any food.
Finally, I was released from the hospital but had to stay
on the steroids for several months in order to wean my body
from the harsh treatment. I could not even look at myself in
the mirror, for my face had swelled up beyond relief.
A little over 1 year later, I found myself in the hospital
again. This time the steroids that I was given did not help. I
kept bleeding and bleeding and suffering, and constantly going
to the bathroom. I was there for over one mo nth of my summer
vacation and lost 25 percent of my body weight. My joints, my
insides, and my psyche were all inflamed. I thought that I
would never leave the hospital that month. It was at that point
that my parents and doctors decided to remove my colon. After a
little while, including 2 years with an ostomy, the physical
pain was gone but the suffering I went through will never leave
me. Several years and several surgeries later my body is, more
or less, back in tact. I wake up everyday and see red lines
running across my stomach, these red lines which represents the
scars from the surgery provide me with a constant reminder of
my struggle with IBD, yet they also serve to remind me of how
lucky I am.
I am lucky because I had access to great physicians and I
was able to have these successful operations and sit before you
today, I hope and pray, physically cured of the disease. But
not everyone with IBD gets to tell a happy ending to their
story. I now spend some of my time working for the Long Island
Chapter of the Crohn's and Colitis Foundation of America,
helping adolescents cope with the disease, on both the
individual and social fronts.
Earlier this year, I visited with a young lady, Ariana
Pappas, the daughter of the CCFA Long Island Chapter President,
on the weekend before her surgery. I could see in her eyes the
fear that she had of the pain and the resulting scars from the
surgery. I also saw the hope that she had that she would be
relieved of this pain if the operation were to be successful.
I often see promising young kids forced to miss months of
school at a time, causing test scores and grades to plummet
from As to Cs. I have seen a young boy ridiculed everyday at
school because his growth has been stunted so significantly by
IBD that he looks 6 years younger than his 13 year old age. I
see children so distraught and so mentally defeated, simply
because they think that talking about the disease is
unacceptable in today's society. Our society has inadvertently
put a cap on bowel discussion, which does not usually become
lifted until those days in the retirement home.
Mr. Chairman, hope for IBD patients and their families lies
in the promise of better treatments and a cure through
biomedical research. To that end, I want to thank Congresswoman
Sue Kelly for her leadership in introducing the Inflammatory
Bowel Disease Act in the House. This landmark legislation,
which addresses IBD research at the NIH and CDC, as well as
other issues that are important to our community, has over 175
bipartisan co-sponsors, including 17 members of this
distinguished subcommittee. On behalf of the CCFA and the
entire IBD community, I want to express our appreciation for
your support, and encourage you to pass this important bill
this year.
In closing, I want to thank you, Mr. Chairman, for your
interest in this disease and for convening this important
hearing today. We are very grateful for your leadership and
look forward to working with you to help improve the quality of
life for IBD patients and their families. I would be pleased
and honored to respond to any questions that you may have.
[The prepared statement of Adam Carron follows:]
Prepared Statement of Adam Carron, Member, Crohn's and Colitis
Foundation of America
Mr. Chairman thank you for the opportunity to testify today. I am
Adam Carron, an 18 year-old ulcerative colitis patient from Long
Island, New York and I appreciate the opportunity to share my story
about living with inflammatory bowel disease with you.
My experience with IBD began at age nine. It started with bloody
stools and intense abdominal pain, and my parents nightmare came true
when my doctor told us all that I probably was suffering from
ulcerative colitis; the same disease that lead to the death of my uncle
at age 42 and the illness my mother was struggling with. Before I could
even pronounce ``Inflammatory Bowel Disease,'' let alone know what it
was, I knew I had something bad and was in trouble. I even feared that
I was going to die. I remember the insecurities affecting my everyday
life; backing out of sleeping at friends houses and going to camp, even
being at school, needing permission to use the bathroom frequently
during the day, fearful of what might happen if the teachers said
``no''. I was given the responsibility to remember to take medicine
everyday, both orally and anally, just so that I would not have stomach
cramps and blood in my stools. I resented it because in a time when all
I wanted was to fit in, none of my friends had the same issues, and
therefore I did not always follow through.
At age 11 I got very sick. The medications were not controlling my
symptoms. I was bleeding profusely and had severe pain. I could not
stop vomiting. I had to go into the hospital. For several weeks I lay
in the hospital, being given massive does of steroids. I'll never
forget having to miss ``Trick or Treating'' with my friends and then
not being able to eat the Halloween candy because I could not have any
food. Finally I was released from the hospital and then had to stay on
the steroids for several months in order to wean my body from the
treatment. I could not even look at myself in the mirror, for my face
had swelled up beyond belief. A little over one year later, I found
myself in the hospital again. This time the steroids that I was given
did not help. I kept bleeding and bleeding and suffering, and
constantly going to the bathroom. I was there for over one month of my
summer vacation and lost over 25% of my body weight. My joints, my
insides, and my psyche were all inflamed. I thought that I would never
leave the hospital. It was at that point that my parents and doctors
decided to remove my colon. After a little while, the physical pain was
gone but the suffering I went through will never leave me. Several
years and several surgeries later my body is more or less back intact.
The physical pain was finally gone but the suffering I went through
will never leave me. I wake up everyday and see red lines running
across my stomach. These scars provide me with a constant reminder of
my struggle with IBD, yet, also serve to remind me just how lucky I am.
I am lucky because I had access to great physicians and I was able
to have these successful operations and stand before you today, I hope
and pray, physically cured of the disease. But not everyone with IBD
gets to tell a happy ending to their story. I now spend some of my time
working for the Long Island Chapter of the Crohn's and Colitis
Foundation of America, helping adolescents cope with the disease, on
both the individual and social fronts. Earlier this year, I visited
with a young lady, Ariana Pappas, the daughter of the CCFA Long Island
Chapter President, on the weekend before her surgery. I could see in
her eyes the fear that she had of the pain and the resulting scars from
the surgery. I also saw the hope that she had that she would be
relieved of her pain, if the operation were successful. I often see
promising young kids forced to miss months of school at a time, causing
test scores and grades to plummet from ``A's'' to ``C's.'' I have seen
a young boy ridiculed everyday at school because his growth has been
stunted so significantly by IBD that he looks 6 years younger than his
13-year-old age. I see children so distraught, and mentally defeated,
simply because they think that talking about the disease is
unacceptable. Our society has inadvertently put a cap on bowl
discussion, which does not usually become lifted until those days in
the retirement home.
Mr. Chairman, hope for IBD patients and their families lies in the
promise of better treatments and a cure through biomedical research. To
that end, I want to thank Congresswoman Sue Kelly for her leadership in
introducing the ``Inflammatory Bowel Disease Act'' in the House. This
landmark legislation, which addresses IBD research at the NIH and CDC,
as well as other issues that are important to our community, has over
175 bipartisan co-sponsors, including 17 members of this distinguished
subcommittee. On behalf of CCFA and the entire IBD community, I want to
express our appreciation for your support, and encourage you to pass
legislation on IBD this year.
In closing, I want to thank you Mr. Chairman for your interest in
this disease and for convening this important hearing today. We are
very grateful for your leadership and look forward to working with you
to help improve the quality of life for IBD patients and their
families. I would be pleased to respond to any questions that you may
have.
Mr. Bilirakis. Thank you so much, Adam, for that very
moving statement.
Let us see, we have three votes on the floor. The first
vote takes a few minutes and then two 5 minute votes after
that. I would like to finish up maybe Dr. Peura's statement and
then we can take a break.
Proceed, sir, and hopefully I will not have to cut you off.
We certainly do not want to miss the first vote.
STATEMENT OF DAVID PEURA
Mr. Peura. Thank you very much.
Mr. Chairman, members of the subcommittee, thank you for
initiating this hearing on assessing digestive disease research
and treatment opportunities, and particularly for allowing the
Digestive Disease National Coalition to present testimony.
I am Dr. David Peura. I am Associate Chief of the Division
of Gastroenterology and Hepatology at the University of
Virginia. I am Professor of Medicine at that institution. I am
also an active clinician, clinical researcher for over 30
years.
Established in 1978, the Digestive Disease National
Coalition is a national non-profit advocacy organization
comprised of the major gastrointestinal volunteer patient
organization and professional societies. Currently there are 25
member organizations that belong to the DDNC. One of the
original members of the coalition is the American
Gastroenterological Association of which I am a member and
currently serve as the President-elect.
The mission of the Digestive Disease National Coalition is
to work cooperatively to improve access to and quality of
digestive health care in order to promote the best possible
medical outcome and quality of life for current and future
patients diagnosed with digestive diseases. The founder of the
DDNC was a Crohn's disease patient who saw the need for
increased digestive disease research and education.
As we have heard, inflammatory bowel disease is just one of
the scores of debilitating gastrointestinal conditions that
afflict more than 62 million Americans. Others include
hepatitis and other liver disease, irritable bowel syndrome,
disease of the pancreas, ulcers, pediatric and adult
gastroesophageal reflux, metabolic disorders, colon cancer,
celiac disease, motility disorders, hemochromatosis and a
number of other serious ailments.
A recent study, ``The Burden of Gastrointestinal Disease,''
conducted by the Lewin Group concluded that a group of just 17
digestive disease disorders accounted for $41 billion each year
in direct and indirect health costs. In some of these areas,
medical research has brought us closer to developing lifesaving
treatments and cures.
For example: The application of immunologic advances which
have made liver transplantation a common life saving approach;
the development of effective screening techniques for
colorectal cancer; the genetic contributions of IBD, which we
have heard about, acute and chronic pancreatitis, pancreatic
and colon cancer and chronic diarrheal illnesses, these are all
just on the cusp of clinical application.
Yet for every breakthrough, there is still a lack of even
basic understanding of the causes, prevention, transmission and
treatments of a variety of other disease.
IBD, the name given to Crohn's disease and ulcerative
colitis, is a painful disrupting disorder, which currently has
no cure. In Crohn's disease, the large and small intestines
become inflamed. This inflammation can result in excessive
diarrhea, severe rectal bleeding, anemia, fever, as well as
abdominal pain and cramping, as we have heard. But I think it
is important to emphasize this for the people that are
afflicted with this condition.
Those battling this disorder face the trauma of multiple
surgeries and the effects of toxic and potentially dangerous
drugs. Ulcerative colitis attacks the large intestine, as we
have heard, causing painful diarrhea, bleeding, and can
ultimately lead to colon cancer, the third most common cancer
among the population in the United States, difficult to
diagnose and often misdiagnosed.
The goal of medical treatment of IBD is to suppress the
inflammation in the large and small intestine, thereby
permitting the intestines to heal and some of the symptoms to
be relieved. While surgery is an option for some people,
removing the segment of the small intestine of the colon, and
may allow people to be symptom free for years, it is not always
a cure.
Mr. Chairman, the Digestive Disease National Coalition
supports the passage of H.R. 290, The Inflammatory Bowel
Disease Act. In addition to being endorsed by the coalition,
the bill is endorsed by 7 of the DDNC member organizations.
The DDNC commends Congresswoman Sue Kelly for her
leadership in introducing this legislation. This bipartisan
bill has 176 co-sponsors, 17 of whom sit on this committee. The
broad support of this bill reflects the tremendous potential in
biomedical research related to IBD. The scientific community,
led by the CCFA, has developed a long range strategic plan for
the future of IBD research and is in agreement that an
additional investment in IBD research has the potential to
yield greater scientific progress in clinical and general
research in other areas as well.
The DDNC also supports the passage of H.R. 3756, The
National Commission on Digestive Diseases Act, introduced by
Congressman Roy Blunt and Congressman Bobby Rush. In 1976,
Congress passed legislation that authorized the first national
commission on digestive disease----
Mr. Bilirakis. Dr. Peura, forgive me, but I am doing
something that I did not want to do. But we have less than 3
minutes left to get over and cast this vote. So you will have
to forgive us. And when we get back, you will finish up. It
will be probably about 20 minutes or so. As soon as we cast the
third vote, we will hustle back here.
Mr. Peura. Fine.
Mr. Bilirakis. Thank you.
[Brief recess]
Mr. Bilirakis. We are back in session.
Dr. Peura, please proceed, sir. And, again, I apologize for
the interruption.
Mr. Peura. Well, actually it was a very good place to
pause, because the presentation is sort of in two halves. We
have covered the issue of the IBD bill.
The DDNC also supports the passage of H.R. 3756, The
National Commission on Digestive Disease Act, introduced by
Congressman Blunt and Congressman Bobby Rush. In 1976, Congress
passed legislation that authorized the first national
commission on digestive diseases. Actually, I am probably
chronologically gifted enough to have practices in the BC era,
and that was before the first commission. And so I have
actually seen the results of that commission.
A quarter of a century later, H.R. 3756 would authorize a
contemporary commission to provide a blueprint for the
digestive diseases research endeavor of the future. Like the
original commission, the new commission would be charged with
assessing the state of digestive diseases in the United States,
identifying areas in which improvement in the management of
digestive diseases could actually be achieved and creating a
long range plan to recommend resources to effectively promote
the GI research portfolio.
We are particularly thankful for the leadership of
Congressman Blunt and Rush on this bill.
In a time of limited fiscal resources to pursue an almost
boundless reservoir of scientific opportunity, it is even more
imperative that the most promising avenues of research are
traveled and that only the highest quality grants, trials,
centers, and other programs are awarded funds. If the work of
the first commission serves as a precedent, this initiative
will once again galvanize the government, the research
community and the expanding population of people suffering from
digestive diseases in a comprehensive and cost effective
national campaign to end the scourge of digestive disorders.
I would again thank you, Mr. Chairman, and this members of
this subcommittee for holding this important hearing, and ask
that the committee pass H.R. 290, The Inflammatory Bowel
Disease Act and H.R. 2756, The National Commission on Digestive
Diseases Act as soon as possible.
And I will be more than happy to answer any questions.
[The prepared statement of David Peura follows:]
Prepared Statement of David A. Peura, Digestive Disease National
Coalition
Mr. Chairman and members of the subcommittee, thank you for
initiating this hearing on Assessing Digestive Disease Research and
Treatment Opportunities and allowing the Digestive Disease National
Coalition and to present testimony. I am Dr. David Peura, Associate
Chief, Division of Gastroenterology and Hepatology and Professor of
Internal Medicine at the University of Virginia Health Sciences Center.
BACKGROUND ON THE DDNC
Established in 1978, the Digestive Disease National Coalition
(DDNC) is a national non-profit advocacy organization comprised of the
major gastrointestinal volunteer patient organizations and professional
societies. Currently there are 25 member organizations that belong to
the DDNC. One of the original members of the coalition is the American
Gastroenterological Association (AGA) of which I am a member and
currently President-elect.
The mission of the Digestive Disease National Coalition is to work
cooperatively to improve access to and the quality of digestive disease
health care in order to promote the best possible medical outcome and
quality of life for current and future patients diagnosed with
digestive diseases. The founder of the DDNC was a Crohn's disease
patient who saw the need for increased digestive disease research and
education.
THE IMPACT OF DIGESTIVE DISEASES
Inflammatory bowel disease is just one of the scores of
debilitating gastrointestinal conditions that afflict more than 62
million Americans; others include hepatitis and other liver diseases,
irritable bowel syndrome, diseases of the pancreas, ulcers, pediatric
and adult gastroesophageal reflux, metabolic disorders, colorectal
cancer, celiac disease, motility disorders, hemochromatosis, and other
serious ailments.
A recent study, The Burden of Gastrointestinal Diseases, conducted
by the Lewin Group, concluded that a group of just 17 digestive
diseases accounts for more than $41 billion each year in direct and
indirect health care costs. In some of these areas, medical research
has brought us closer to developing lifesaving treatments and cures.
Examples of this include:
The application of immunologic advances which have made liver
transplantation into a common life saving approach.
The development of effective screening techniques for colon cancer.
The genetic contributions to IBD, acute and chronic pancreatitis,
pancreatic and colon cancer and chronic diarrheal diseases
which are just on the cusp of recognition.
Yet for every breakthrough, we still lack even a basic
understanding of the causes, prevention, transmission and treatments
for other diseases.
IBD, the name given to Crohn's disease and ulcerative colitis, is a
painful and disrupting disorder, which currently has no cure. We see in
Crohn's disease the large and small intestines have become inflamed.
This inflammation can result in excessive diarrhea, severe rectal
bleeding, anemia, fever, as well as abdominal pain and cramping. Those
battling this disorder have the trauma of multiple surgeries and the
effects of toxic and potentially dangerous drugs. Ulcerative colitis
attacks the large intestine, causing painful diarrhea, bleeding, and
can ultimately lead to colon cancer, the third highest cancer
population in the United States.
IBD is an unpredictable disorder, symptoms vary in nature,
frequency, and intensity. I wish I could say IBD was an easy disease to
diagnose, but it is not. Misdiagnosis is common. Because there is no
cure for IBD, the goal of medical treatment is to suppress the
inflammation of the large and small intestine and the colon. By
suppressing this inflammation, intestinal and colon tissue is permitted
to heal and relieve many symptoms. Surgery can be an option to remove
the diseased segments of the bowel or the colon. While surgery might
allow patients to be symptom-free for many years, it is not a cure.
LEGISLATIVE INITIATIVES IN DIGESTIVE DISEASES
Mr. Chairman, the Digestive Disease National Coalition supports the
passage of H.R. 290, The Inflammatory Bowel Disease Act. In addition to
being endorsed by the coalition, the bill has been endorsed by many of
the DDNC member organizations.
The DDNC commends Congresswoman Sue Kelly (R-NY) for her leadership
in introducing this legislation. This bipartisan bill has 176 co-
sponsors, 17 of which sit on this committee. The broad support of the
bill reflects the tremendous potential in biomedical research related
to IBD. The scientific community, led by CCFA, has developed a long-
range strategic plan for the future of IBD research and is in agreement
that an additional investment in IBD research has the potential to
yield greater scientific progress in clinical and general research.
The DDNC also supports the passage of H.R. 3756, The National
Commission on Digestive Diseases Act, introduced by Congressman Roy
Blunt (R-MO) and Congressman Bobby Rush (D-IL). In 1976, Congress
passed legislation that authorized the first National Commission on
Digestive Diseases. The Commission was charged with assessing the state
of digestive diseases in the United States identifying areas in which
improvement in the management of digestive diseases could be achieved,
and creating a long-range plan to recommend resources to effectively
deal with promoting the GI research endeavor. The Commission, because
it provided a credible roadmap for research and generated enthusiasm
within the biomedical community, precipitated a number of research
breakthroughs.
In a time of limited fiscal resources to pursue an almost boundless
reservoir of scientific opportunity, it is all the more imperative that
the most promising avenues of research are traveled and that only the
highest quality grants, trials, centers, and other programs are awarded
funds. If the work of the first Commission serves as precedent, this
initiative will once again galvanize the government, the research
community, and the expanding population of people suffering from
digestive diseases in a comprehensive and cost-effective national
campaign to end the scourge of digestive disorders. Like its
predecessor, the Commission should be directed to develop and recommend
a long-range plan for the use and organization of national resources to
effectively deal with digestive diseases.
I would again thank you Mr. Chairman and the members of this
subcommittee for holding this important hearing and ask that the
committee pass H.R. 290, The Inflammatory Bowel Disease Act and H.R.
3756, The National Commission on Digestive Diseases Act as soon as
possible.
Mr. Bilirakis. Thank you very much, Mr. Peura.
I do not know whether any other members will return. Please
do not think that there is a lack of interest in the subject;
there is not. But it is a very hectic, frantic place,
obviously, and a lot of other obligations and that sort of
thing. Mr. Brown particularly extends his regrets because I
know there is a group from Cleveland who--I guess they purchase
from a charity, they purchase the right to have lunch with him
or something. I think the Rotary Club or something of that
nature. It is a critical that he has got to go. And so I know
he apologizes.
I am just going to ahead.
For some time, Drs. Spiegel and Peura particularly, going
back to the prior Administration in the sense that when the
Democrats controlled the Congress and whatnot, we on this
committee felt that we should not be determining what specific
dollars should be earmarked for what specific disease. You
know, the feeling was that the people at NIH know where the
breakthroughs might be pretty darn close and, therefore, they
are in a much better position if you will, to determine how
many dollars should go to--Sue and I have talked about this.
And so anyhow, it has always been our policy to not set out
specific amount of dollars that will be going to research for a
specific disease.
We had doubled NIH funding. We had planned to continue to
increase NIH funding. And we have been hoping that they would
do the job adequately in terms of allocations of dollars.
I mean, I have had Mohammad Ali come in here on behalf of
Parkinson's and plead for more funding for Parkinson's. We can
go on, ALS, Alzheimer's. You can just go on and on. And, of
course, every group considers their disease, if you will, the
highest priority and I do not blame me. So, you know, what can
we really do here? So that has always been sort of our feeling.
And I am going to ask for the two of you particularly, what
your opinion is regarding that? Do you think it is right for
Congress to decide what diseases deserve more attention than
others or is this priority setting activity, as I have already
said, best determined by the scientists and advisory counsels
at the NIH.
Dr. Spiegel?
Mr. Spiegel. Thank you, Mr. Chairman.
Let me just say that I again want to emphasize how
enormously appreciative we are both of your leadership and the
bipartisan support in Congress that permitted the doubling of
the NIH budget and allowed us to initiate some of these
important digestive disease initiatives that I alluded to.
The reality is that science is extraordinarily complex.
Because it is so complex, it is difficult often to predict
where the next discovery is coming from. I gave the example of
a diabetes drug which targets a receptor that turns out to be
very highly expressed in the colon, and actually reduces
inflammation in the colon first in animal models and then in
the clinical trial, and which we are studying extensively and
may become a therapy for ulcerative colitis. So for this reason
we really do appreciate the flexibility in being able to set
priorities.
Having said that, and having met with your staff in April,
it is vital for the NIH priority setting process to be as
transparent as possible. I am committed to doing the best
possible job we can in making that as transparent as possible.
And we do that by inviting and sharing with stakeholders such
as the CCFA, the AGA, that we are hearing from, from patient
groups, traveling around the country meeting with patient
groups. Again, as an example, the CCFA has crafted a tremendous
strategic plan in IBD which was then extensively presented at
an April 2003 meeting that Dr. James chaired at our Digestive
Disease Interagency Coordinating Committee. This was an
opportunity for all the NIH institutes to hear this plan and
work toward its implementation.
The bottom line ultimately is that, given the complexity of
science, given the interrelation between things that you work
on in one area that end up being very helpful to another area
and the rapid pace with which these things change, we
appreciate the flexibility to be able to set priorities, but in
no way do we have any sense of arrogance or being isolated in
saying that we are going to do this in a top down way. It has
got to be transparent, it has got to have input from external
groups and stakeholders, and that is what we are committed to.
Mr. Bilirakis. Should it have in effect mandates from
Congress in terms of the amount of dollars that should be
allocated to that particular disease?
Mr. Spiegel. Let me not be presumptuous. We are fortunate
to live in an extraordinary democracy and you as the elected
representatives of the people ultimately are responsible to
your constituents. I view myself, as a physician, scientist and
public servant, as accountable to you as the elected
representative and want to work in partnership with you in each
of these areas. To the extent that you offer us the flexibility
to do this priority setting and do it in the most transparent
way possible, we certainly appreciate that. And I know that Dr.
Zerhouni as the NIH Director, who has interacted extensively
with you, does. This gives us the opportunity.
I will cite just one other example. There are many areas of
both basic fundamental research as well as large projects. Take
the human genome project. That was an enormous undertaking of
the NIH. It was just the first step in a way, just getting all
those 3 billion A, G, C and Ts, but it was the investment in
that which was not specific to any disease that then allowed a
tremendous acceleration in the discovery of the Crohn's
susceptibility gene. That would not have happened without the
investment in something that in this case was not disease
specific. We have to keep that perspective in mind.
I know you have heard from Dr. Zerhouni about the NIH
Roadmap. In my many ways that is another example; the idea of
increasing the harmonization of clinical research practices.
That has got to be a benefit to people with IBD, with ALS, with
every disease where we are desperate for better treatments and
preventions, and where accelerating clinical trials and really
being sure that we have the public's input is vital.
Mr. Bilirakis. So you feel that you have adequate
flexibility?
Mr. Spiegel. We never take it for granted and are
appreciative of having that flexibility.
Mr. Bilirakis. Dr. Peura?
Mr. Peura. Well, again, I want to take the opportunity
again to thank you for having this hearing and allowing me to
testify.
Mr. Bilirakis. Is the mic on?
Mr. Peura. It is a very difficult question you ask. I mean,
as a clinician I see people with lots of different disease, but
I know that inflammatory bowel disease, for example, what we
are discussing here is a disease that causes significant
morbidity, mortality. And we have heard from Adam about the
morbidity that it caused him.
Money spent for a particular disease is not just for that
disease. And as Dr. Spiegel had mentioned, there are amazing
outcomes that come from understanding immunology that cross
many, many different disease states. Much of what we would
learn by research in IBD would also be applicable to arthritis
and neuro-degenerative disorders and other sorts of things. It
is really not to say that anything is disease specific, because
the fundamentals of research cross many disease entities.
I do know that digestive diseases as a class of disease
causes significant morbidity, mortality. And as I mentioned, I
practiced in the BC era. Before really--and usually when I say
that it is before cimetidine which was really probably one of
the first advances in pharmacology in gastroenterology where we
treated ulcer disease. Endoscopy was based on ulcer disease.
Colostomy was based basically on ulcer disease, but now we
recognize that ulcer disease is an infectious disease and
research going in there, now we can cure a chronic disease. We
spent a lot of money in the past on ulcer, but we recognize
that the advances in endoscopy, colostomy, surgical techniques,
all of that came from ulcer disease.
So, I am obviously an advocate for money for digestive
disease, but I think money is well spent on disease like IBD
because of its implications for a variety of conditions.
Mr. Bilirakis. You both said it well, but I think you said
it more like a lawyer than you did--but you know, we have these
good people, people like Adam coming and testifying here and
wanting us, almost pleading with us--he has not done so, but
really pleading with us that X amount of dollars, should be
more funding for, as I said, Parkinson's or funding for ALS.
And Sue has dollars in her particular piece of legislation that
would be earmarked specifically for these diseases, and
whatnot.
So, you know, again the question is I mean what do we tell
them? I cannot explain it the way you all have. What do we tell
them? It is right for Congress to decide what diseases deserve
more attention than others; and that is the question. And I
think you both said in a sense that it is not right that
Congress do that. At least I think Dr. Spiegel has said that in
a round about way? It's important because we are met--you know,
we do not have to meet with the patients just as you do. But
they are pleading with us. Yes, they are pleading with you to
get them well. They are pleading with us to allocate more
money, which we can hope of course that more money would result
in a cure. I mean, there is a hope thing there.
Anything further?
Mr. Peura. Well, Mr. Chairman, I mean I think that if I
were answering that question and somebody came into my office
and said that, I would probably say I want to be as committed
to supporting research. Research in one particular area, as we
have mentioned, is going to effect the disease that you have,
the disease that other people have, too. The problem is when we
do not fund research, when we shut the faucet off and, you
know, research suffers and many generations of physicians
suffer. I mean, I am faced with young physicians everyday that
are making a decision whether they should have a research
academic career or whether they should go into private
practice. Private practice is vital. There have to be people
out here taking care of the Adams in this world. But there also
have to be people, the best and the brightest, that are
available to apply for the NIH funding and for the other
funding, CCFA funding that is out there.
And to say, to sort of support that by being very positive
in research and targeting those areas such as IBD that really
will have an opportunity to make a major impact, not only on
lives of sufferers of IBD, but patients with a variety of
digestive conditions and nondigestive. I mean, I think that
would be my answer, that I am committed to research. And I
think it is important to have prioritization, but commitment to
research and research that is going to bear fruit.
As I said, following the best and most productive avenues I
think is going to be important. And that is what a digestive
disease commission would also help do.
Mr. Bilirakis. Mr. DeRose, I believe it was you who
accented maybe a lack of cooperation, lack of funding from CDC.
Was it not you that made that comment? Yes. Why do you not
expand on that?
Mr. DeRose. For some time now the Appropriations Committee
has had dollars allocated for an epidemiology study at the CDC,
but those dollars have not been spent or focused in the area of
IBD epidemiology study. And our frustration as an organization
from a patient point of view has been that if the allocation is
there and we can make a strong case as a patient group on the
needs of this growing dilemma that we have in our country, why
cannot those dollars be allocated appropriately by the CDC to
move this research forward.
And so our frustration stems from that. And to the point
where we said we are going to put our money where our mouth is,
and that is what we believe it is important. And we will go to
the CDC and ask them to submit a grant, have it peer reviewed
through the same process that we use for all our grants. And if
it passes, and is approved, then we will fund dollars to it.
But we felt it important enough that we take a lead on it
on behalf of being able to come to you and say, Mr. Chairman,
it's not a million Americans; it is 2-, it is 3-, it is 4
million Americans.
Mr. Bilirakis. We do not really know.
Mr. DeRose. We do not know. The last study was done in 1991
in Olmstead, Minnesota. And that, to me, is not the best
representation of every aspect of the country, nor every ethnic
group of the country. And there are some high incidents of this
disease in ethnic groups.
So I would say that that was our frustration with the CDC.
And we still have a very positive relationship with them, and
it will continue. But since they report to you as a
stakeholder, I think it is incumbent on you and the
Appropriations Committee to ask penetrating questions with the
knowledge that you have to be able to say why are we not
funding, why are we not proceeding with this study that has
already been allocated.
Mr. Bilirakis. Okay. And you will be available for any
inquires we may have to help us?
Mr. DeRose. Sure.
Mr. Bilirakis. Good.
Adam, you were at the CCFA event on Long Island, were you
not? You were not there?
Mr. Carron. I do not believe I was at that particular gala.
Mr. Bilirakis. You were not at that particular one?
Mr. Carron. I do not think--I am actually positive I was
not there.
Mr. Bilirakis. Well, I was there. And I started to get
tears in my eyes during your testimony, and I guarantee I had
them when she, of course, had something like a half hour to
share her experiences with us. And, you know, the feeling was
at the time that she was basically well. She had improved so
tremendously, and then she went to camp, and you have told me.
What has happened since she went to camp, tell us?
Mr. Carron. She had her first surgery, which was at Mount
Sinai, and it was supposed to be her final surgery.
Mr. Bilirakis. Yes, she had had many surgeries. Well, she
had surgeries over a period of years.
Mr. Carron. Yes. I am sorry. This surgery at Mount Sinai
was supposed to be her final surgery.
Mr. Bilirakis. Right.
Mr. Carron. And it was very similar to the operation that I
had where they would take the small intestine and form a pouch
basically and then remove the colon. And so kind of reconnect
that there.
But while she was at camp, she had an obstruction which
formed by the scar tissue on the inside which had basically
closed up and she was forced to come from camp and go back into
the operating room to have that basically reopened up. And I
believe she is currently recovering from that right now.
Mr. Bilirakis. Yes. Well, I notice that you counsel a lot
of these young people, adolescents and whatnot on this disease.
Are these young people who have the disease?
Mr. Carron. Yes. I am serving as kind of the co-director of
youth activities for the Long Island Chapter. And through that
I am--I get deferred to a lot of younger kids. And I will go to
the hospital to visit them or call them, and basically kind of
like relax them and let them know what they are about to go
through and to share my experiences with them so that--to that
basically so that they will be able to deal with the IBD as
best as they can and feel most confident doing so.
Mr. Bilirakis. Well, you are to be commended, Adam, for
your courage, for your toughness, for your willingness to come
here and share with us. And even though there are not other
members other than Ms. Kelly here to ask our questions, a
record is being taken. And I assure you it will all be very,
very helpful.
I have taken more than my time, because not only be the
Chair but also because nobody is here to do it, but I will
yield to Ms. Kelly for her inquiry.
Ms. Kelly. I thank you, Mr. Chairman. I thank you so much
for letting me here and be a part of this hearing today. I
really appreciate your generosity on that.
Mr. DeRose, I want to go back to the issue about the
support that CCFA has given the Centers for Disease Control. As
I understand it, the CDC has not provided any support for IBD
epidemiology program, that program.
The IBD Act includes the provision that will formally
establish the epidemiology program, and it is really needed.
Mr. Chairman, I think it is important that we recognize
organizations that contribute their own resources to projects
that benefit the general public good and to promote the types
of public/private partnerships in the way that the CCFA has
stepped forward to do. This is an unusual organization. The
members of CCFA have demonstrated that they are willing to fund
research, they have put a lot of money in research and in
education. And I just want to go on record as saying I feel
very strongly that we in Congress owe it to organizations like
that to try to at least match the amount that they have
collected and put into the research on their own because it is
the type of research, as both the doctors on this panel have
pointed out, will extend the life of many people from many
different kinds of diseases. Autoimmune disease are a very
particularly interesting group of diseases. And bits of
research can be put together to help others all the way along
the line.
And the other thing I like to just go on record as saying
is this Congress does fund specific diseases. We fund research
in AIDS, we fund research in cancer and we just passed a bill
to fund research on stroke. It seems to me if we do that for
those three diseases where we have such a strongly committed
group of people who have collected such large amounts of money
on their own and offered it to the CDC, we in Congress really
could help a lot of people if we would at least be able to
match that by requesting of the CDC that amount of money.
I do not know, Mr. DeRose, if you would like to--I mean, I
welcome this opportunity to ask you question. I would like to
hear if you have got any more thoughts on this, because I think
this is extraordinary? I do not know of other groups that--you
are not a large groups, you are a small group and you have
collected a lot of money and you have put a lot of research
grants out there on your own. You have done a lot just to try
to build the information base. And that is really what, I
think, in part you are asking for now. And I would just like to
point out that that is the reception that I got from you, and I
want to know if you would like to just respond to that?
Mr. DeRose. Well, Congresswoman, I would be echoing some of
the points that you have made and think I mentioned earlier. I
really do think that it is important for organizations such as
ours to take a leadership role. If a government agency is not
going to take action on an objective that has been laid out for
them, then we are. And we will make the investments so that we
can show to them, as well as to the community, that we are in
position to advance knowledge and advance information so that
we can get the true picture of this disease into the right
hands so that you can make those kinds of decisions that are
needed.
I personally feel that we as a disease state should be
getting more dollars allocated to this disease. It is a complex
disorder. It is not a one gene type of a disease, it is
multiple genes. It is probably, depending on the literature, 10
to 20 genes that affect this disease state and we have only
discovered one. And there is great activity that is taking
place right now in other chromosomes. Chromosome 5 and 10 that
I think once verified, and it is being verified now in
independent labs around the world, we may be on the
breakthrough of other genes that it may behoove us for further
understanding----
Mr. Bilirakis. Well, but if the gentleman will yield. But
do you not make that case to NIH? You made that case to CDC,
and apparently they have not been cooperative. But you make
that case.
I mean, who are we to make that case to? Who are we to
understand the breakthrough and the complexities, as you
indicated, and the technicalities and whatnot, whereas those
people are the experts? And as long as we give them the
funding, it seems like you should be able to make the case to
them and then, hopefully, the funding would be adequate.
Mr. Spiegel. If you will permit me, I would like to
interject something which is relevant to this point and
actually harks back in a way to what Mr. Buyer I believe had
said in some of his initial statements.
We work with the CCFA in a unique kind of public/private
partnership which I really value, and I think that Congressman
Kelly has underscored.
Mr. Bilirakis. I think it is terrific.
Mr. Spiegel. In a sense they are providing seed money to
investigators who would not be able to get NIH grants without
preliminary data. This is the system that we have. And it is a
conservative system, but it has to be because currently under
the best of circumstances only one of three applications can be
funded. The reality is that we have to be extremely circumspect
and we want some preliminary evidence that they can accomplish
what they are setting out to do.
It is the CCFA and organizations like it, and there are
some other organizations in our institute that we work with
this way, who provide that seed money. As you heard from Mr.
DeRose, it then leverages multifold because of the generous
resources you have given us.
That is one dimension that I think is very important, and I
really do value that partnership.
Let me just illuminate, and this comes to Mr. Buyer's point
earlier, that we want to be extremely circumspect with every
hard earned tax dollar that you give us in terms of how it is
used. We do not want to reinvent the wheel. We do not want to
do the work of private industry. We are not talking about
nonprofit voluntary groups such as the CCFA, but industry in
terms of the pharmaceutical and biotech industry. In that
respect we are really now very much focused on what we call a
translational emphasis for inflammatory bowel disease. We are
spending at NIH as a total for fiscal year 2003 of $58.4
million on IBD. That money, though, is really targeted to areas
in which we believe only NIH, only government support could
really make a difference. Let me just illuminate that.
We led an almost groundbreaking kind of meeting with the
FDA in January of 2003. Dr. James led that meeting, and
organized that meeting. The drug industry and biotech industry
were there trying to work out ways to get better indices of the
disease. There's something called the Crohn's disease activity
index, which is a very crude measure of disease. The fact is
that you have to constantly do endoscopy, and Dr. Peura is an
expert he knows how to do this, but it is invasive in a way. We
need ways that are noninvasive, a simple blood test for example
that could tell us how bad this disease is. These are the kinds
of things, fundamental investments, which industry cannot do
but which we can do; we have set these out as important goals.
Finally, just as an example, there is an interesting
situation vis-a-vis ulcerative colitis versus Crohn's disease.
There are actually over 10 or more new agents in the pipeline
at varying stages short of approval, some of which will come
through, for Crohn's disease. There is lesser activity,
actually, in ulcerative colitis. Why is that? The fact is that
you have this drastic maneuver of colectomy, removing the
entire large bowel as a ``cure.'' It is an alternative, but it
is not the desirable outcome that we want. We want to be able
to avoid that with effective kinds of treatments. For that
reason we have focused extensive attention on ulcerative
colitis. With this diabetes drug that I mentioned, there is no
guarantee that is going to work, but we will not find out
unless we are supporting that kind of effort.
Another example is the a drug azathioprine. It is a generic
drug which is an immunosuppressant. It is now generic so there
is no patent exclusivity and there is less incentive for drug
companies to really be working or testing that. Again, Dr.
James' leadership is involved in funding studies based at the
University of Chicago and other places that are looking at the
metabolism of that drug. There are wide differences among
children and others in terms of the metabolism of that drug so
that we need to be able to know what is the most effective dose
that will suppress the disease and yet not have dangerous side
effects. This is the kind of work that only NIH can support,
and this is why we are so grateful for the support you are
giving us to focus our attention on these areas and the
translational opportunities.
Ms. Kelly. Reclaiming my time.
Dr. Spiegel, I would like to follow up with you on that
because I am not really clear right now. From what you said it
seems to me that we need to do the epidemiologic study that the
CCFA has asked for. We have not gotten the support for that
study from the CDC. And what I do not understand is are you are
talking about a lot of things. There is a lot of things other
there. But what I do not understand is why if NIH and CDC are
connected, there cannot be some pressure from NIH on CDC to get
that epidemiologic study done.
Mr. Spiegel. It is an excellent point. I can tell you, I am
an ambitious guy, and I think an energetic guy, and I find that
running my institute gives me a full time job as opposed to
necessarily running other parts of the CDC. We are sensitive to
what you are saying. We value colleagues at the CDC and we
actually work with them in a variety of areas, often in very
close partnership.
The physician and epidemiologist, I think her name is
Siobhan O'Connor, at the CDC has been involved in conducting
this study. She is someone we have had up to the NIH to
Bethesda. We have wanted to hear from her about her methods. We
are in dialog about these kinds of things. Maybe you will
accuse me of not being sort of forceful enough, but I have not
seen it as within my purview to dictate what they should be
doing in regard to this study.
Ms. Kelly. Well, I am just a little puzzled about the
money. We are giving NIH a lot of money, CDC has a lot of
money. Crohn's and Colitis Foundation has given CDC a lot of
money. And there is no movement on the part of CDC.
Mr. Chairman, I am wondering if perhaps it would be helpful
to the CCFA if you and I wrote a letter directly to the CDC and
asked them why there is no movement on that and just weigh in
on the fact that it might be----
Mr. Bilirakis. We have been known to do that. And I have
already talked to Cheryl about it. That is why I made the
comment about maybe asking Mr. DeRose would be available when
we might need some information toward that.
Yes, we will follow up as far as that is concerned.
Your time has basically expired.
Ms. Kelly. Thank you.
Mr. Bilirakis. Look, we want to do the right thing, okay.
We sort of ask, you know, put yourselves in our shoes
sometimes, too. You know everybody, I mean there is never
enough money, No. 1. There certainly is not ever enough money
for any of the diseases, and yet the squeaky wheel gets the
grease and there have been some diseases that the wheels have
been more squeaky than others and have gotten funding, mostly
through the appropriation process. But we do want to do the
right thing. And we also have to realize that money--money is
significant as far as research is concerned, but there are
other ways in addition to--in addition to, not in lieu thereof
but obviously in addition to things that Adam have shared with
us that he is doing with some of the younger people and things
of that nature could be helpful.
So, to adjourn the hearing, but I would like to invite you,
and I mean it sincerely and I do it every hearing, is to feed
into us--well, first of all we are going to have a series of
questions to you that we will submit to you in writing and we
are requesting that you respond to them in a timely fashion,
number. Particularly as it might involve a piece of
legislation.
I am hopeful that before this year is up we are going to
have some legislation on this issue. I cannot tell, I am not
all powerful enough to tell Bobby Rush that his legislation
will become law, and I am not powerful enough to tell Sue that
her legislation will become law. But I am hoping that we will
have a good piece of legislation that will combine some of the
better parts of both pieces of legislation that, hopefully,
will be set up in such a way that they will get the proper
credit for it. Because that is important. They do not do it for
credit, but I think it is obviously important that that be
taken into consideration.
So taking that into consideration, respond to our questions
as soon as you can. Feed into us any additional points you want
to make that you have not made here today that might come to
mind, any suggestions you may have to crank into the
legislation, things of that nature. Let us not fight the battle
of what this President did or what the previous President did,
or anything of that nature. I mean, that is the wrong way to
go. That is what really gets us off the track when we get into
this partisanship business, and it happens unfortunately much
too much up here. And we throw stones at each other rather than
get our heads together and do what is right for the public out
there. And you saw a little bit of an idea of that earlier
today. And that is unfortunate. But I do know that all of these
people are concerned, and I do know that they ultimately want
to do the right thing. It is how we go about it sometimes that
is maybe not the right way to go.
Adam, you come up with any ideas; first of all, if you see
Ariana, give her my best, but also any ideas that you might
have that would be helpful to us in order to be able to do a
better job, please do not hesitate.
Thank you so very much, gentlemen.
The hearing is adjourned.
[Whereupon, at 1:07 p.m. the hearing was adjourned.]
[Additional material submitted for the record follows:]
Response for the Record by David A. Peura, Digestive Disease National
Coalition
Q. As has been stated, Congressman Blunt and I have introduced a
bill that would create a temporary commission to conduct research on
digestive diseases. Historically, Congress has established similar
research commissions on many disease areas. Can you please comment on
why it makes sense for Congress to act and create a Digestive Diseases
Research Commission?
A. During the late 1990s, digestive diseases researchers and
clinicians reported that there was a growing incidence in the number of
patients with serious gastrointestinal diseases. In an effort to gain
external validation for this growing problem, the AGA contracted with
the Lewin Group in 2001 to conduct a study entitled ``The Burden of
Gastrointestinal Diseases.'' The study focused on just 17 of the scores
of digestive diseases and included a comprehensive analysis of at least
5 nationally recognized health statistics data bases to determine the
overall annual financial burden, in direct and indirect costs,
associated with these 17 diseases. The study found that the combined
direct and indirect costs to society for these diseases in 2000 were
$42 billion. While the study only focused on a subset of all digestive
diseases, the results have been recognized as substantive documentation
that the U.S. faces a crisis in terms of the incidence of digestive
diseases.
A Digestive Diseases Research Commission would focus the research
community on setting priorities and research goals and address these
issues in a trans-NIH and trans-scientific community fashion. This
mechanism has been recognized as a successful model by scientists for
over 35 years, with the first Digestive Diseases Research Commission in
the 1970s as a prime example. The commission would be required to
report back to Congress to ensure accountability in its deliberations.
Q. The first Digestive Diseases Research Commission was created
back in the 1970s. Dr. Peura, can you comment on the benefits of
creating a second Research Commission on digestive diseases?
A. A second commission would provide an important opportunity to
update the work of the very successful first Digestive Diseases
Research Commission. Approximately 15-18 commissioners would be
appointed by the Secretary of HHS, drawing from the ranks of
distinguished scientists in digestive and other diseases,
representatives from research in other federal agencies, patient
advocates, the pharmaceutical industry, technology experts, and
industry leaders impacted by the loss of productivity related to
digestive diseases. Their efforts would concentrate on the following:
Developing a comprehensive forward-looking strategic plan for
addressing the crisis in digestive diseases over the next 5-10
years;
Addressing overarching approaches to new and effective clinical
trials, specific clinical studies, refining new technologies
and setting standards in these areas;
Recommending more collaborative, inter-institute research and ways to
accelerate new initiatives to more quickly and efficiently find
effective treatments and cures;
Assessing the need for more researchers in digestive diseases to meet
the challenges posed by the growing incidence of digestive
disorders;
Updating the work of the first commission due to the advent of
genomics and genetics research, new technologies and the
discovery of new digestive diseases and how to apply this
information to new research initiatives.
The efforts of the first commission led to the advent of powerful
new therapies such as interferons and monoclonals and new technologies
such as CT colonography and capsule endoscopy. The discovery of the H.
pylori bacteria as the cause of ulcers and the IBD 5 gene's role in
Inflammatory Bowel Disease are also major breakthroughs set in motion
by the first commission. The first commission also recommended the
establishment of the Digestive Disease Core Research Centers program
which remains a model of collaborative research even today.
We still face many new challenges in the area of obesity, hepatitis
and hepatomas, and the potential use of foodborne pathogens as
bioterrorism agents just to name a few of many existing diseases and
threats.
Q. Some critics of our bill contend that a Digestive Diseases
Research Commission duplicates NIH functions already in place, such as
the work done by the National Institute of Diabetes and Digestive and
Kidney Diseases. Would you please tell us how a temporary research
commission would supplement the work of the NIDDK and its advisory
committee?
A. Actually, the NIDDK Advisory Council, which meets 3 times per
year for approximately 1\1/2\ days for each meeting, operates much
differently than a commission. The Council is comprised of 18 members,
only 2 of which are experts in gastrointestinal diseases. The primary
function of the Council is to provide second level peer review for
current research grants, grant issues and budget issues (i.e. the size
of grants) which occupies approximately 50% of their meeting time. The
other portion of the meeting is occupied with updates on ongoing NIH
programs. As such, the NIDDK Advisory Council, as with other institute
councils, does not address strategic issues or long-range research
planning, but more of the ``here and now'' operational issues. It also
does not operate as an inter-institute body. The Council serves an
important operational role for the NIDDK but has a clearly distinct
mission from that of a commission.
It should also be noted that one of the major outcomes of the
advisory council process, scientific ``consensus conferences'' on
specific diseases, reflect a culmination of research and not new
research and, as a result, are not intended to develop new knowledge. A
Digestive Diseases Research Commission would complement the Advisory
Council's work by providing a forward-looking mechanism for developing
a long-range plan to help accelerate research into more effective
treatments and cures for digestive diseases.
Q. Can you tell us the differences between creating an internal
research commission and an external commission as Mr. Blunt and I are
proposing? What are the merits and/or disadvantages of each approach?
A. An internal commission would impose upon NIH to create a
potentially permanent structure which is currently not in place. The
logistics of creating such a body would be cumbersome, potentially
disruptive of the current work being undertaken by scientists and
administrators and impractical as it would require individuals to
change roles and methods of operating for a defined period of time. NIH
might also be required to hire more full time staff to accommodate an
internal commission and, thereby, create a more permanent and costly
alternative to an external commission. Digestive diseases research is
also such a broad area of investigation that it lends itself more to
community participation than internal deliberation. Only 10% of all
research monies are designated to intramural research so the depth of
expertise does not exist within the structures of NIH. Finally, due to
the restrictions placed upon federal agencies, an internal commission
exercise would be confined to supporting the parameters of the
President's budget request and would not be conducive to creative
thinking and long-range planning, thereby being more reactive in
nature. In addition, the effort would not be a trans-NIH initiative
and, therefore, would not be a centerpiece of other institutes'
programs.
An external commission would provide an opportunity for academic
gastroenterologists and other specialists to provide leadership and
accept responsibility for the elements of a comprehensive long-range
plan addressing digestive diseases. This exercise would also energize
the digestive diseases community. Since 90% of NIH research is not
conducted in Bethesda, but elsewhere in the country, it is essential to
energize, and achieve buy-in, from the community that ultimately will
implement the plan. This approach would also be the most effective in
helping ensure any initiatives included in the plan are trans-
scientific community and trans-institute.